Hope for Sickle Cell Warriors: A Cure Exists and Support from the Black Community Could Help Save More Lives – EBONY

When Constance Benson* was 25 years old, she was living her dream as a model with a major agency. One day, while receiving treatment for a pain crisis in the hospital, she realized she wasnt well enough to travel any longer so she gave up her modeling dream. As a sickle cell warrior, Constance had already left college because she was unable to keep up with school work as a result of her unpredictable pain crises.

She was uncertain of her future. She recalls feeling tormented by the response of medical professionals who doubted the severity of her condition with one nurse telling her she was too pretty to be sick. Constance considered it divine intervention when she learned that a blood stem cell or marrow transplant could cure her from sickle cell disease. Her parents are both nurses, but it wasnt until her mom was caring for a patient whose son had just had a transplant to cure his sickle cell disease that she learned this was even a treatment option.

To date, hundreds of people in the USA and around the world have been cured of sickle cell disease following blood stem cell or marrow transplants from compatible donors. Like Constance, too often people are unaware that transplant is even an option.

A new day has arisen for sickle cell disease treatment. For the first time, increasingly more medical and research efforts are directed at better understanding and treating sickle cell disease. Historically, progress in developing new treatments for sickle cell disease had been slow. This has been in part due to the complexity of sickle cell disease itself and underfunding compared to other potentially life-threatening genetic diseases.

Among children under the age of 16 battling sickle cell disease, about 95% were cured from sickle cell disease after a transplant from a matched sibling. A blood stem cell or marrow transplant from a matched sibling is now considered a pediatric standard of care by many doctors, and transplant from matched unrelated donors are becoming more common.

Too often people suffering from severe sickle cell disease are unaware of treatments that exist and may be available to them. For example, clinical trials that offer cutting-edge treatment options like blood stem cell or marrow transplantation may be an appropriate path for someone who is not responding well to prescribed therapies. Clinical trials are highly regulated and administered by medical experts. They are designed to assess the safety and effectiveness of new treatments. A benefit of clinical trials includes access to specialized care for your condition with built-in safety measures.

With a transplant, blood stem cells or marrow is extracted from a compatible donor and given to a sickle cell disease patient via IV. The donors healthy blood-forming stem cells, which create healthy red blood cells, replace a patients unhealthy stem cells that made sickled red blood cells. For the person with sickle cell disease, a successful transplant can mean no more sickled red blood cells with no more blocked blood vessels and no more damage from ruptured cells. Some people will even see existing damage improve with the introduction of healthy blood stem cells. Risks do exist for transplant, but doctors can inform a patient about the benefits and risks of such a procedure so that the patient and their family can make an informed decision about proceeding.

Transplant too often is dismissed as an option for people with sickle cell disease by themselves, or by their families, or even by their physicians because of concerns that the financial cost would be prohibitive. There are financial resources available to assist families considering transplant as an option, so it should not be ruled out as a treatment for a person battling advanced sickle cell disease.

As representatives from Be the Match and the Sickle Cell Transplant Advocacy and Research Alliance (STAR), we want to encourage people with sickle cell disease and their loved ones to be empowered to educate themselves about new treatment options, to ask questions of their primary care or hematology providers and to consider participating in clinical trials if other treatments are not working.

Constances younger sister was a perfect match for her. Today, Constance is living free from sickle cell disease after a successful marrow transplant seven years ago. She was able to return to college and obtained her bachelors degree, and she is committed to spreading awareness of the need for more diverse donors on the Be The Match Registry.

Nearly 1 out of 5 people with sickle cell disease will find a match within their family. Those without a match in their family turn to Be The Match, the national marrow donor program, to try to find an unrelated donor willing to help a complete stranger find their cure.

When searches are performed for Black patients, there is a 23% chance of finding a compatible donor on the Be The Match Registry. This is because race and ethnicity play a role in finding a matched blood stem cell or marrow donor. The makeup of each of our cells is as diverse as the places our grandparents and ancestors came from and people of African descent have more unique and complex genes than other races. Currently, only 4% of the 20 million donors on the worlds largest registry are Black or African American. Given all the diversity among persons of African descent, more donors are needed.

This underrepresentation can be improved by more Black donors joining the registry and it only takes a cheek swab to get started. Its important to note that not only people with sickle cell disease, but patients battling blood cancers like leukemia or serious blood disorders like aplastic anemia, also are searching for matches on the registry. There is an urgent need for more racially and ethnically diverse donors to join the registry. Visit http://www.endsicklecell.org to learn how.

A diagnosis of sickle cell disease can mean frequent emergencies, life threatening infections, irreversible organ damage, and even early death. Sickle cell disease frequently denies patients and families what any of us would want comfort, time, growth and financial stability. Sickle cell disease causes excruciating acute pain in children and, as patients age, the pain becomes chronic and debilitating for adults. STAR is comprised of hematologists and supporters of the sickle cell community who are dedicated to advancing research that will help lead to a cure for sickle cell disease. We have partnered with Be The Match to ensure that people with sickle cell have access to free resources, including information about clinical trials, access to certified nurses and patient navigators to learn more about transplant as an option, and a new Peer Connect program that will match existing patients with sickle cell transplant recipients. For more information, call the Be The Match Patient Support Center at (888) 999-6743 or visit http://www.bethematch.org/sicklecell. To learn more about research initiatives and success stories being supported by STAR, visit http://www.curesicklenow.org.

Currently, the only cure for sickle cell is a blood stem cell or bone marrow transplant, but new methods of gene therapy are now also being tested. With more than 100,000 persons with sickle cell disease in the U.S. today, the need for safe and effective treatment options and the need for more donors is high.

We hope that EBONY readers will take action to help address sickle cell disease in the Black community:

Disclosure: Constance Benson and her family have given consent to Be The Match to share her story.

In the USA, approximately 1 in 365 people of African descent will be born with sickle cell disease, an inherited blood disorder where red blood cells are abnormal. Healthy red blood cells are soft and oval-shaped as they travel throughout the body delivering oxygen to organs. Red blood cells that contain sickle hemoglobin can become stiff and crescent-shaped. Sickled cells block blood flow, causing excruciating pain, lung damage and potential strokes. They also rupture, releasing debris that causes damage to blood vessels.

Some people with sickle cell disease manage symptoms as they come. Other patients take a daily medication that decreases the likelihood of forming sickled red blood cells. Still others might receive chronic red blood cell transfusions to reduce the number of sickled cells in their bloodstream. Additional patients choose to participate in clinical trials to pursue new treatments that offer a chance at a cure. Visit http://www.Clinicaltrials.gov to learn about qualifying to participate in a trial.

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Hope for Sickle Cell Warriors: A Cure Exists and Support from the Black Community Could Help Save More Lives - EBONY

Latest Report on Stem Cell Therapy Market To Flourish And Reach USD 4759.27 Million By 2024 – News Hours Today

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Latest Report on Stem Cell Therapy Market To Flourish And Reach USD 4759.27 Million By 2024 - News Hours Today

Ruth Lehmann elected as director of Whitehead Institute – MIT News

The Whitehead Institute board of directors today announced the selection of Ruth Lehmann, a world-renowned developmental and cell biology researcher, as the institutes fifth director. Lehmann will succeed current Director David Page on July 1, 2020.

Lehmann is now the Laura and Isaac Perlmutter Professor of Cell Biology and chair of the Department of Cell Biology at New York University (NYU), where she also directs the Skirball Institute of Biomolecular Medicine and The Helen L. and Martin S. Kimmel Center for Stem Cell Biology. She is currently an investigator of the Howard Hughes Medical Institute. The Whitehead Institute appointment represents a homecoming: Lehmann was a Whitehead Institute member and a faculty member of MIT from 1988 to 1996, before beginning a distinguished 23-year career at NYU.

Ruth Lehmann will continue a line of prestigious and highly accomplished scientist-leaders who have served as Whitehead Institute directors, says Charles D. Ellis, chair of the Whitehead Institute board of directors. She perfectly fits our vision for the next director: an eminent scientist and experienced leader, who is passionately committed to Whitehead Institutes mission, and possesses a compelling vision for basic biomedical research in the coming decade.

I am delighted to return to Whitehead Institute and look forward to joining the illustrious faculty to recruit and mentor the next generation of Whitehead Institute faculty and fellows, Lehmann says. When I was recruited to Whitehead Institute in the late 1980s, David Baltimore took a huge risk in giving an inexperiencedyoung scientist from Germany the chance to follow her passion for science with unending encouragement and minimal restraints. Now I am thrilled to have the opportunity to help shape the future of this wonderful institute that has been at the forefront of biomedical research for decades. I am pleased to become part of the succession of Whitehead Institutes forward-thinking directors, David Baltimore, Gerald Fink, Susan Lindquist, and David Page. I look forward to working with faculty, fellows, trainees, and staff to build a future with ambitious goals that will allow us to reveal the unknown and connect the unexpected in a collaborative, diverse, and flexible environment.

Ruth Lehmann is an inspired choice to lead the institute into the future and I look forward to working with her in that capacity, Page says. Ruth is an internationally renowned and influential leader in the field of germ cell biology, and her outstanding contributions to the field are the product of her sustained brilliance, insatiable curiosity, uncompromising rigor and scholarship, and clarity of thought and expression.Across the course of the past three decades, no scientist anywhere in the world has made greater contributions to our understanding of germ cells and their remarkable biology. Im especially pleased to gain a colleague with such an impressive track record of discovery and institutional leadership.

The new director will have an impressive line of predecessors: Whitehead Institutes founding director was Nobel laureate and former Caltech president David Baltimore; he was succeeded by internationally honored geneticist and science enterprise leader Gerald Fink, and then by National Medal of Science recipient Susan Lindquist, followed by the current director, leading human geneticist David Page, who became director in 2004.

Ruth Lehmann is a brilliant choice as the next director of Whitehead Institute, Baltimore says. She is a world-class scientist and a seasoned leader. Most importantly, she understands the unique nature of Whitehead Institute and will maintain it as a key element of the biomedical complex that has grown up in Cambridge, Massachusetts.

Ruth Lehmann is an extraordinary scientist, who began her distinguished career here at Whitehead, Fink says.Her innovative work on germ cells, which give rise to eggs and sperm, has paved the path for the entire field. She is an inspiring leader who is an outspoken advocate for fundamental research.We are all delighted to welcome her back as our new director and scientific colleague.

Lehmann has made seminal discoveries in the field of developmental and cell biology. Germ cells, the cells that give rise to the sperm and egg, carry a precious cargo of genetic information from the parent that they ultimately contribute to the embryo, transmitting the currency of heredity to a new generation. Work in Lehmanns lab using Drosophila (fruit flies) has shed light on how these important cells know to become germ cells, and how they are able to make their way from where they originate to the gonad during early embryonic development. Her discoveries uncovering the mechanisms needed for proper specification and migration of germ cells have not only informed our understanding of processes essential for the perpetuation of life itself, but have also made important contributions to related fields including stem cell biology, lipid biology, and DNA repair.

I'm so pleased to be welcoming Ruth back to the community, MIT Provost Martin A. Schmidt says. Her dedication to, and expertise in, basic research will underscore Whitehead Institute's reputation as a leader in this arena.

Susan Hockfield, MIT president emerita and professor of neuroscience, chaired the committee that recommended Lehmann to the Whitehead Institute board. Our committee considered eminent candidates from across the globe, Hockfield says, and found in Ruth Lehmann a person uniquely qualified to guide this pioneering research institution forward.

Lehmann earned an undergraduate degree and a PhD in biology from the University of Tubingen in Germany, in the laboratory of future Nobel laureate Christiane Nsslein-Volhard. Between those programs, she conducted research at the University of Washington and earned a diploma degree equivalent to a master's degree in biology from the University of Freiburg in Germany. She then conducted postdoctoral research at the Medical Research Council Laboratory of Molecular Biology in Cambridge, England. Then, Lehmann moved to Cambridge, Massachusetts, to become a Whitehead Institute member and MIT faculty member. In 1996, she accepted a professorship at NYU Langone School of Medicine and was subsequently named director of the Skirball Institute of Biomolecular Medicine and The Helen L. and Martin S. Kimmel Center for Stem Cell Biology, NYU Stem Cell Biology Graduate Program director, and chair of the NYU Department of Cell Biology in 2014 (all roles that she continues to hold).

She has served as president of the Society for Developmental Biology, the Drosophila Board, and the Harvey Society; is currently editor-in-chief of the Annual Review of Cell and Developmental Biology; and will serve as president of the American Society for Cell Biology starting in 2021. Additionally, she has been a council member of the National Institute of Child Health and Human Development.

Among her many awards, Lehmann has received the Society for Developmental Biologys Conklin Medal, the Porter Award from the American Society for Cell Biology, and the Lifetime Achievement Award from the German Society for Developmental Biology. She is an elected member of the National Academy of Sciences, a fellow of the American Academy of Arts and Sciences, and a member of the European Molecular Biology Organization.

Lehmann has also been a committed mentor, having fostered the education and professional development of scores of undergraduate and graduate students and postdoctoral researchers. Many of her mentees have gone on to become leaders in the biomedical industry or at academic institutions in the United States and around the world, including Johns Hopkins University, Princeton University, MIT, the University of Cambridge (UK), European Molecular Biology Laboratory (Heidelberg, Germany), and University of Toronto (Canada).

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Ruth Lehmann elected as director of Whitehead Institute - MIT News

Regulatory heft needed to curb false promises on stem cells, says health law expert – Folio – University of Alberta

The legal and regulatory tools designed to protect the public from the marketing of unproven stem cell therapies will remain ineffective without bureaucratic will and grassroots efforts, according to a University of Alberta health law expert.

There's this perception that stem cells are revolutionizing science and they have transformed medicine already, but that's just not the case, said Timothy Caulfield.

You see the word stem cells being used to sell everything from skin cream to sports recovery tools to supplements, it's absolutely everywhere.

Caulfield, who refers to the marketing of spurious stem cell treatments as scienceploitation, explained there are actually only a handful of such therapies that have been approved for use in a clinical setting.

The most well known is probably the use of stem cells in bone marrow transplants and certain kinds of leukemiabut these therapies have been around for decades, he said, adding other stem cell therapies have shown some effectiveness in the treatment of bad burns and blindness.

But that's it.

In a paper outlining a strategy to combat the spread of misrepresentation within this field, Caulfield and Health Law Institute research associate Blake Murdoch argued the first step is to leverage the powers wielded by the provincial colleges of physicians and surgeons.

We need a more robust response from them because they have the power to stop their members from marketing treatments inappropriately and from offering services that are unproven, said Caulfield.

We haven't seen that, and it really is their role to protect the public.

He added organizations aimed at stopping the spread of misinformation and inaccurate marketinglike Ad Standards, Canadas advertising industry's non-profit self-regulating body, or Competition Bureau Canada, the federal advertising regulatorcan also be more involved.

While the Competition Bureau can only prohibit clinics from using misleading advertising and

not the provision of unproven interventions, this would help to stop the spread of misinformation, which may curtail public interest, said Caulfield.

He added political pressure on federal and provincial lawmakers could encourage change and allow a more comprehensive response, but noted that targeting the marketing of these treatments might be the more politically palatable course of action.

I think a really good logical first step is if you're going to market this stuff, if you're going to offer these services, the information you're using to market the services has to be accurate.

Even as the paper was being published, Caulfield said Health Canada weighed in by stating stem cell therapies need its approval.

Basically, if youre an MD, and you're providing stem cell therapy, you need to get it approved, said Caulfield. In the paper, we said Health Canada has got to get more aggressive, and thankfully, we're starting to see some action in that space.

He said ultimately, however, responses from Canadas regulatory bodies are often triggered by complaints from the public.

I've actually spoken to regulators, and theyre not hearing complaints about people being injured by stem cell treatment, said Caulfield. Of course, just because something is safe, doesn't mean it's a good idea.

Not only have some of these treatments shown to have caused real harm while offering little more than hope, Caulfield said there is a financial exploitation element, all of which can only leave a black mark on the science.

The spread of clinics marketing these interventions may, over the long term, damage public trust in legitimate regenerative technologies, thus adversely impacting their future development, he said. It confuses what is an incredibly promising field.

The most perplexing element of the proliferation of these treatments is the involvement of medical professionals who should know better, Caulfield said.

The team went into the analysis with the hypothesis that alternative practitioners were the ones providing and marketing stem cell therapies. This was true, but Caulfield said they were surprised to find that an MD was often involved.

I've been in the room with these health providers, and you get the sense that many of them believe it works.

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Regulatory heft needed to curb false promises on stem cells, says health law expert - Folio - University of Alberta

Releases New Report on the Stem Cell Banking Market 2017-2025 – Rapid News Network

The stem cell banking is the practice of preserving blood cell from a new born baby for future use. The preserved blood is used in medical therapies for a variety of diseases, which include conditions such as sarcoma, leukemia, immune conditions, lymphoma, and metabolic disorders. Stem cells are capable of producing blood cells, namely red blood cells, platelets, and white blood cells, which are produced in the human body. Researchers have discovered that stem cells can be used for the treatment of 80 different diseases including thalassemia, sickle cell anemia, leukemia, diabetes, and cardiac diseases. The major sources for stem cell bank are storing of peripheral blood and bone marrow, umbilical cord blood (UCB) is by far considered as the largest component for hematopoietic stem cells. In the past few years, donated umbilical cord blood has become a reliable source of stem cells, and is thus considered as a valuable biological resource. The chances of cord blood sample matching with the patients that is closely in relation to the baby whose sample is to be used are 39% more than the sample obtained from the public banks. Stem cell banking services is a promising and fast growing segment in the field of next generation stem cell therapy and national cord blood registries are changing perception and look of the industry.

Rise in incidence of chronic conditions and increase in investments by pharmaceutical and biopharmaceutical companies for stem cell research are expected to drive the global stem cell banking market during the forecast period. Rise in the number of stem cell donors and superior quality of stem cell banking facilities and increase in research & development are expected to drive the global stem cells market. However, high processing & storage cost and lack of acceptance and awareness in developing regions are likely to inhibit the market.

The global stem cell banking market can be segmented based on source, service type, application, end-user, and geography. In terms of source, the stem cell banking market can be categorized into placental stem cells, adipose tissue-derived stem cells, bone marrow-derived stem cells, human embryo-derived stem cells, dental pulp-derived stem cells, and others. Based on service type, the stem cell banking market can be divided into sample preservation and storage, sample analysis, sample processing, and sample collection & transportation. In terms of application, the stem cell banking market can be classified into research applications, clinical applications, and others. Based on end-user, the global stem cell banking market can be segmented into hospitals (medical applications), pharmaceutical research (drug discovery), research institutes (scientific research), and others.

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In terms of region, the global stem cell banking market can be classified into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. In terms of revenue, North America dominated the stem cell banking market in 2017, followed by Europe. North America dominated the global market due to increasing prevalence of chronic diseases and expanding network of stem cell banking services. The market in Asia Pacific, Latin America, and Middle East & Africa is anticipated to expand at a high growth during the forecast period due to growth in the health care industry and increase in patient population in these regions. The market in China, Brazil, and India is projected to expand at a considerable pace between 2018 and 2026 due to rise in investment by governments of these countries to improve health care facilities.

Key players operating in the global stem cell banking market include Cord Blood Registry (CBR) Systems, Inc., Cryo-Cell International, Inc. Cordlife Group Limited, Lifecell International Private Limited, Cryo-Save AG, and StemCyte.

The report offers a comprehensive evaluation of the market. It does so via in-depth qualitative insights, historical data, and verifiable projections about market size. The projections featured in the report have been derived using proven research methodologies and assumptions. By doing so, the research report serves as a repository of analysis and information for every facet of the market, including but not limited to: Regional markets, technology, types, and applications.

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The regional analysis covers: North America (U.S. and Canada) Latin America (Mexico, Brazil, Peru, Chile, and others) Western Europe (Germany, U.K., France, Spain, Italy, Nordic countries, Belgium, Netherlands, and Luxembourg) Eastern Europe (Poland and Russia) Asia Pacific (China, India, Japan, ASEAN, Australia, and New Zealand) Middle East and Africa (GCC, Southern Africa, and North Africa)

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Releases New Report on the Stem Cell Banking Market 2017-2025 - Rapid News Network

In Mice and Men, Prostate Drug Reportedly Treats Parkinson’s Disease? – Alzforum

20 Sep 2019

A drug commonly prescribed to keep enlarged prostates in check may hold promise for Parkinsons disease. According to a study published September 16 in the Journal of Clinical Investigation, terazosin (TZ), an 1-adrenergic receptor antagonist that moonlights as a glycolysis booster, raised ATP, upped mitochondrial numbers, and ultimately saved dopaminergic neurons from degeneration in multiple models of PD. Led by Michael Welsh at the University of Iowa in Iowa City and Lei Liu of Beijing University, the researchers claim that among men who took terazosin or related drugs to quell prostate hyperplasia, the incidence of PD was low, and symptoms of diagnosed disease were mild.

The researchers uncovered TZs energy-boosting prowess serendipitously, Welsh explained. In Beijing, TZ popped up as a top hit in Lius screen for cell death inhibitors in fruit flies. Flies lack 1-adrenergic receptors so, probing further, Liu found that the drug activates phosphoglycerate kinase 1 (PGK1), one of two enzymes that generate ATP in the glycolysis pathway (Chen et al., 2015). Since mitochondrial malfunction and impaired bioenergetics are features of withering neurons in PD, the researchers decided to test this ATP-boosting drug in models of the disease.

First author Rong Cai and colleagues began with the MPTP injection mouse model of parkinsonism. Given daily at the time of injection of this mitochondrial toxin, TZ attenuated all core pathologies, including the drop in ATP, loss of mitochondria in the striatum, loss of dopaminergic neurons, and loss of balance. When the researchers delayed TZ treatment until seven days after MPTP injection, the drug still had beneficial effects on dopaminergic function and motor performance by day 14.

TZ similarly protected other toxin models of parkinsonism, including 6-OHDA in rats, and rotenone in flies. In the insects, knocking out PGK1 erased the benefits of TZ, bolstering the idea that the drug fends off a PD-like syndrome via this enzyme.

Shrinking Both Prostate and Synuclein? In iPSC-derived neurons from Parkinsons patients, -synuclein (green) accumulates in dopaminergic neurons (red). TZ treatment reduced accumulation to control neuron levels. [Courtesy of Cai et al., JCI, 2019]

In genetic models of Parkinsons, the researchers saw TZ treatment assuage motor deficits, for example in flies carrying PD mutations in PINK1 or LRRK2. In transgenic mice overexpressing wild-type -synucleinthe primary component of the Lewy body inclusions that define the diseasetreatment with TZ between three and 15 months of age partially protected against loss of balance. Finally, in induced pluripotent stem-cell-derived neurons from two PD patients who carried the G2019S mutation in the LRRK2 gene, TZ increased ATP levels and lessened accumulation of-synuclein aggregates (see image above).

Could TZ really work for people with PD? Because the drug is commonly prescribed for benign enlargement of the prostate in men over the age of 60an age group at risk for PDWelsh reasoned that database sleuthing could begin to answer this question. The researchers first turned to the Parkinsons Progression Markers Initiative database, which tracks symptom progression in people with PD. They identified seven men with PD who took TZ and 269 who did not. Men on TZ had a slower rate of motor decline. Still in the PPMI database, the researchers expanded their query to include related drugsdoxazosin (DZ) and alfuzosin (AZ)which are also used to treat prostate enlargement and contain the same quinazoline motif shown to enhance PGK1 function. The 13 men taking TZ, DZ, or AZ had slower progression of motor decline than those not on the drugs. Crucially, 24 men with PD who took tamsulosin, another prostate drug that antagonizes 1-adrenergic receptors but does not activate PGK1, did not have this relative protection. I just about fell off my chair when I saw those results, Welsh said.

Drugs Help Pee and PD? Men with PD who took TZ, DZ, or AZ had a lower relative risk of 69 of 79 PD-related diagnoses in their charts compared to men who took tamsulosin. Yellow dots represent a statistically significant difference. Diagnoses are grouped into categories. [Courtesy of Cai et al., JCI, 2019]

To expand their search to more people, the researchers accessed the IBM Watson/Truven Health Analytics MarketScan Database for insurance claims relating to PD. They identified 2,880 men with PD who took TZ, DZ, or AZ, and 15,409 men with PD who took tamsulosin. They next selected 79 diagnostic codes related to PD, such as falls, tremor, walking problems, and sleep disorders. They found that compared with men with PD who took tamsulosin, those taking TZ, DZ, or AZ had a 22 percent lower relative risk of having any of these diagnostic codes in their files, suggesting they had milder disease. They also had fewer hospital visits for motor and non-motor symptoms, as well as PD complications.

To see if the ATP-boosting drugs might delay or prevent PD, the researchers identified more than 78,000 men in the Truven database without PD who took TZ, DZ, or AZ, then tracked their files for 284 days. During that time, 118 of them were diagnosed with PD, compared with 190 age-matched men who took tamsulosin instead. This yielded a hazards ratio of 0.62, suggesting that TZ, DZ, and AZ reduce the incidence of PD.

All of this is very encouraging, and indicates that TZ is a strong candidate for clinical trials to see if it can be repurposed for Parkinsons, wrote Chris Elliott of the University of York, U.K. In this TZ joins other drugs (including UDCA and Exenatide) that affect energy metabolism. Howeve, Elliott noted that the mechanism of TZs effects on neurons remains to be ironed out, and cautioned that the drug has risks. It reduces blood pressure, which may already be low in people with Parkinsons, and so careful evaluation of its safety is needed."

Clemens Scherzer of Brigham and Womens Hospital in Boston noted that defective bioenergetics is a key pathway in Parkinson's, and that the study points at potential tool compounds to correct these defects. The quinazoline 1 adrenoreceptor blockers, which are used for benign prostatic hyperplastia, could be repurposed for clinical trials in PD or chemically tweaked to develop brain-optimized, new bioenergetics drugs for PD, Scherzer told Alzforum. He added that rigorous population-wide cohort studies and clinical studies will be needed.

Welsh told Alzforum that it is unclear exactly how TZ and related drugs might counteract PD. He noted that recent studies indicate that ATP itself interferes with -synuclein aggregation and liquid phase separation (Patel et al., 2017;Hayes et al., 2018). ATP could also reduce protein aggregation by bolstering the activity of myriad enzymes, including heat-shock proteins, he added. Of course, ATP might enhance all manner of neuronal functions by supplying cells with more energy. Welsh is investigating whether the drugs affect progression or incidence of other neurodegenerative proteinopathies, including AD.

Welsh has initiated a small trial to test TZ in PD patients at Iowa University, and has applied for funding to get multicenter trials up and running. He is starting by dosing patients with 5 mgas prescribed for prostate enlargementbut said dose-finding studies are needed. Complicating matters, the drug had a biphasic dose response on its PGK1 target in cultured cells and in mice, meaning that at higher concentrations, it no longer elevates ATP. Welsh also noted that though the primary use of the drug is for prostate enlargement, it was also briefly used to treat hypertension, and was tested in women as well as men for that indication. He plans to include both men and women with PD in trials.Jessica Shugart

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In Mice and Men, Prostate Drug Reportedly Treats Parkinson's Disease? - Alzforum

Stem Cell-Derived Cells Market to Record an Exponential CAGR by 2025 – Commerce Gazette

Stem cell-derived cells are ready-made human induced pluripotent stem cells (iPS) and iPS-derived cell lines that are extracted ethically and have been characterized as per highest industry standards. Stem cell-derived cells iPS cells are derived from the skin fibroblasts from variety of healthy human donors of varying age and gender. These stem cell-derived cells are then commercialized for use with the consent obtained from cell donors. These stem cell-derived cells are then developed using a complete culture system that is an easy-to-use system used for defined iPS-derived cell expansion. Majority of the key players in stem cell-derived cells market are focused on generating high-end quality cardiomyocytes as well as hepatocytes that enables end use facilities to easily obtain ready-made iPSC-derived cells. As the stem cell-derived cells market registers a robust growth due to rapid adoption in stem cellderived cells therapy products, there is a relative need for regulatory guidelines that need to be maintained to assist designing of scientifically comprehensive preclinical studies. The stem cell-derived cells obtained from human induced pluripotent stem cells (iPS) are initially dissociated into a single-cell suspension and later frozen in vials. The commercially available stem cell-derived cell kits contain a vial of stem cell-derived cells, a bottle of thawing base and culture base.

The increasing approval for new stem cell-derived cells by the FDA across the globe is projected to propel stem cell-derived cells market revenue growth over the forecast years. With low entry barriers, a rise in number of companies has been registered that specializes in offering high end quality human tissue for research purpose to obtain human induced pluripotent stem cells (iPS) derived cells. The increase in product commercialization activities for stem cell-derived cells by leading manufacturers such as Takara Bio Inc. With the increasing rise in development of stem cell based therapies, the number of stem cell-derived cells under development or due for FDA approval is anticipated to increase, thereby estimating to be the most prominent factor driving the growth of stem cell-derived cells market. However, high costs associated with the development of stem cell-derived cells using complete culture systems is restraining the revenue growth in stem cell-derived cells market.

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The global Stem cell-derived cells market is segmented on basis of product type, material type, application type, end user and geographic region:

Segmentation by Product Type Stem Cell-Derived Cell Kits Stem Cell-Derived Definitive Endoderm Cell Kits Stem Cell-Derived Beta Cell Kits Stem Cell-Derived Hepatocytes Kits Stem Cell-Derived Cardiomyocytes Kits Accessories

Segmentation by End User Hospitals Research and Academic Institutions Biotechnology and Pharmaceutical Companies Contract Research Organizations/ Contract Manufacturing Organizations

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The stem cell-derived cells market is categorized based on product type and end user. Based on product type, the stem cell-derived cells are classified into two major types stem cell-derived cell kits and accessories. Among these stem cell-derived cell kits, stem cell-derived hepatocytes kits are the most preferred stem cell-derived cells product type. On the basis of product type, stem cell-derived cardiomyocytes kits segment is projected to expand its growth at a significant CAGR over the forecast years on the account of more demand from the end use segments. However, the stem cell-derived definitive endoderm cell kits segment is projected to remain the second most lucrative revenue share segment in stem cell-derived cells market. Biotechnology and pharmaceutical companies followed by research and academic institutions is expected to register substantial revenue growth rate during the forecast period.

North America and Europe cumulatively are projected to remain most lucrative regions and register significant market revenue share in global stem cell-derived cells market due to the increased patient pool in the regions with increasing adoption for stem cell based therapies. The launch of new stem cell-derived cells kits and accessories on FDA approval for the U.S. market allows North America to capture significant revenue share in stem cell-derived cells market. Asian countries due to strong funding in research and development are entirely focused on production of stem cell-derived cells thereby aiding South Asian and East Asian countries to grow at a robust CAGR over the forecast period.

Some of the major key manufacturers involved in global stem cell-derived cells market are Takara Bio Inc., Viacyte, Inc. and others.

The report covers exhaustive analysis on: Stem cell-derived cells Market Segments Stem cell-derived cells Market Dynamics Historical Actual Market Size, 2014 2018 Stem cell-derived cells Market Size & Forecast 2019 to 2029 Stem cell-derived cells Market Current Trends/Issues/Challenges Competition & Companies involved Stem cell-derived cells Market Drivers and Restraints

Regional analysis includes North America Latin America Europe East Asia South Asia Oceania The Middle East & Africa

Report Highlights: Shifting Industry dynamics In-depth market segmentation Historical, current and projected industry size Recent industry trends Key Competition landscape Strategies of key players and product offerings Potential and niche segments/regions exhibiting promising growth A neutral perspective towards market performance

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Global & US Gene Therapy Market 2019-2020: Immunodeficiency Syndromes – The Focus of Gene Therapy – PRNewswire

DUBLIN, Sept. 20, 2019 /PRNewswire/ -- The "Global & US Gene Therapy Market Forecast to 2020" report has been added to ResearchAndMarkets.com's offering.

Gene therapy is one of the most widely researched fields in the healthcare industry. The high potential of gene therapy to cure various chronic diseases makes it a popular research area. Various researches are being performed across the globe to explore the potential of gene therapy for the treatment of incurable diseases, such as diabetes, cancer, and HIV amongst others.

According to this new research report "Global & US Gene Therapy Market Forecast to 2020, most of the gene therapy researches are being focused on finding the treatment for cancer, followed by genetic diseases and neurological disorders, respectively. In this context, the gene therapy application chapter of the report provides a comprehensive overview of various diseases in which the gene therapy is used, along with the current and future market size of gene therapy for particular disease and its geographical break up. Primarily, the gene therapy market is dominated by oncology applications, with several companies and academic institutions focusing on novel and difficult to treat' cancers. Other therapeutic areas seeking developments in gene therapy include monogenic diseases, cardiovascular diseases, infectious diseases, inherited blindness and neurological diseases. Furthermore, the chapter also provides details regarding the various aspects of the clinical trials in the different gene therapy application by phases, vector types, etc.

The major part of the revenue of gene therapy market is generated from research phase. Most of the gene therapy products are in research phases, only few products have been commercialized till date. The report also provides the sales of major marketed gene therapy products, and the list of the products in clinical/pre-clinical research along with their clinical phases.

The companies operating in the gene therapy market are also receiving various funding, grants, and investment from government bodies and venture capitalist firms which are aiding them to develop new products. The study highlights the applications for which the investments have been received.

North America continues to have the maximum number of clinical trials in the gene therapy segment. This is a major reason for the dominant position of North America in the gene therapy market. Based on the geography, the market is divided into four regions, namely, North America, Europe, Asia, and Rest of the World. The report provides the market for each of the geography, along with its forecast till 2020.

Gene silencing, advanced therapies combining gene therapy and stem cell technology, immunodeficiency syndromes, growing interest of venture capital firms, etc. will propel the industry's growth. An analysis has also been done of a few factors limiting the growth of the industry. The report also provides insights regarding the strategies adopted by the players from 2013 to 2015 for enhancing their market share. Finally, with a view to understand the competitive landscape, the profiles of key market players have been included in the report to present a complete picture of the global gene therapy market.

Key Topics Covered:

1. Analyst View

2. Research Methodology

3. Gene Therapy - An Introduction3.1 Classification of Gene Therapy Techniques3.2 Physical Methods of Gene Transfer3.2.1 Electroporation3.2.2 Hydrodynamic3.2.3 Microinjection3.2.4 Particle Bombardment3.2.5 Ultrasound-Mediated Transfection3.3 Vectors for Gene Therapy3.3.1 Viral Vectors3.3.1.1 Adenoviral Vectors3.3.1.2 Adeno-associated Virus Vectors3.3.1.3 Retroviral Vectors3.3.1.4 Lentiviral Vectors3.3.2 Non Viral Vectors3.3.2.1 Naked DNA/Plasmid Vectors3.3.2.2 Oligonucleotides3.3.2.3 Liposomes, Lipoplexes and Polyplexes3.3.2.4 Gene-Activated Matrix

4. Industry Overview4.1 Market Drivers4.1.1 Failure of Conventional Therapies to Treat Cancers4.1.2 Rising Focus to Accelerate Commercialization of Gene Therapy in Developed Nations 4.1.3 Rising Incidence of Chronic and Life-Threatening Diseases4.1.4 Growing Interest of Venture Capital Firms4.2 Market Restraints4.2.1 Stringent Regulatory Laws & Safety Concerns 4.2.2 Challenges in Commercialization of Gene Therapy4.2.3 High Cost of the Gene Therapy Drugs4.2.4 Pitfalls in Current Technique4.2.5 Production & Manufacturing: A Unique Challenge for Gene Therapies 4.3 Market Opportunities4.3.1 Approval of Gene Therapy Drug in Europe4.3.2 Gene Therapy for Hemophilia B Offers Significant Opportunity 4.4 Industry Trends4.4.1 Gene Silencing: Gaining Momentum4.4.2 Advanced Therapies: Combining Gene Therapy and Stem Cell Technology4.4.3 Immunodeficiency Syndromes: The Focus of Gene Therapy4.4.4 Nanotechnology: Empowering Gene Therapy4.4.5 Gene Therapy: A New Hope to Treat Blindness4.4.6 Gene Therapy: Potential Cure for Cancer4.5 Winning Imperatives4.5.1 Investment in R&D Activities

5. Clinical Trial Assessment & Pipeline Analysis5.1 Clinical Trials5.1.1 By Geography5.1.2 By Indication5.1.3 By Gene Type5.1.4 By Vector5.1.5 By Clinical Trial Phase5.2 Pipeline Analysis

6. Gene Therapy Market - Regulatory Landscape & Reimbursement Scenario6.1 Regulatory Landscape6.1.1 US6.1.2 Canada6.1.3 Europe 6.1.4 Japan6.1.5 China6.1.6 India6.1.7 Australia6.2 Reimbursement Scenario in Global Market

7. Marketed Gene Therapies7.1 Neovasculgen7.2 Glybera7.3 Gendicine, Rexin-G, Oncorine7.3.1 Gendicine7.3.2 Rexin G 7.3.3 Oncorine

8. Gene Therapy Market8.1 Global8.2 US

9. Gene Therapy Market by Application9.1 Oncology9.1.1 Clinical Trials 9.1.2 Prevalence9.1.3 Market Outlook9.2 Cardiovascular Diseases9.3 Infectious Diseases9.4 Neurological Diseases9.5 Genetic Diseases9.6 Others

10. Gene Therapy Market Size by Geography10.1 North America10.2 Europe10.3 Asia10.4 Rest of the World (RoW)

11. Competitive Landscape11.1 Strategies Adopted by Various Players11.1.1 Strategic Collaborations11.1.2 Acquisitions 11.1.3 Funding & Investments11.2 Companies Progress in Gene Therapy In 2015

12. Key Players Analysis (Business Description, Clinical Pipeline, Recent Developments, Strengths & Weaknesses)12.1 Spark Therapeutics, LLC12.2 ViroMed Co. Ltd. dba VM BioPharma12.3 Advantagene Inc.12.4 Bluebird Bio12.5 Sanofi12.6 Vical Inc.12.7 Oxford BioMedica Plc12.8 Genethon12.9 uniQure N.V.12.10 Human Stem Cells Institute12.11 Shanghai Sunway Biotech Co. Ltd.12.12 Sibiono GeneTech Co. Ltd.

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Global & US Gene Therapy Market 2019-2020: Immunodeficiency Syndromes - The Focus of Gene Therapy - PRNewswire

Ten-Year Bone Cancer Survivor Rides ‘Coast 2 Coast’ – Curetoday.com

Dana Vaughns and his colleagues rode 3,000 miles across the U.S. in support of funding research for the V Foundation.

BY Kristie L. Kahl

I just knew that if there was a way to get through (my cancer) and help inspire some others along the way, then I was all for it, Vaughns said. Here I am 10 years later, just completing the bike ride, which is something that I never thought I'd be able to do especially even just riding a bicycle in in the position that I would be in on a consistent basis, putting that stress on the area where I had my surgery to treat the bone cancer was a concern. But now I'm strong enough to be able to get through it and I'm very, very proud to have been able to complete it.

CURE spoke with Vaughns about his experience riding in the Coast 2 Coast 4 Cancer ride and why events like this are important to continue funding research for cancer treatments with the V Foundation.

CURE: What was running through your mind leading up to the event?Vaughns: Anxiety, a little nervousness thinking, Can I actually complete this? I mean, you could say that all the way up until the point of Now youve got to go and make it happen. And when I was out there with my other colleague, going up those hills and needing some support and some energy, I would look up, I would look at a name on someone's shirt, or even think about the names that are on my shirt that I was riding on behalf of, that really propelled me to get up every single hill that was in front of us.

Knowing what it is that I was riding for and who I was riding for, there was no way I was going to stop. I had to do it on behalf of them, I had to do it on behalf of myself. I knew that if I could complete this, then that would be a triumph over cancer for me. And for those that I was riding for, whether I was riding for them in honor of or in support of what they're going through currently right now. So, it was really just more of an inspiration for me to get through this process. And I knew that if I can do it, and those that are still here that are currently fighting cancer, they can overcome anything and that was that was a major goal for me.

What did crossing the finish line mean to you?Wow, it was it was exhilarating. I mean, to have completed 200 miles, to have climbed over 10,000 feet, to have sat on a bicycle saddle for 14 hours, to have realized that all of the training leading up to this point now had come to a conclusion was exhilarating. It was something that I'll never forget for sure.

Why do you think events like this are important?It's important because we ensure that we're staying in front of this thing called cancer. And if we can continue to rally local support, national support, do the fundraising efforts from the ground from the grassroots level in to raise funds for innovation. It's the right thing to do. And we have to find a way to be able to come up with solutions for cancer, whether they be cured, whether they be better treatments, we just have to ensure that we stay out there because the next person that may be diagnosed is going to rely on that they're going to need that. Our kids and our kids kids need to be doing this today. It is very important that we're not just doing this for now. But we're doing it for tomorrow. And that's why I think it's critically important for us to continue to do the resources necessary that can help someone when diagnosed at some point their in their lives. Know that they have a solution for what they've got going on. I think that's really critical for us to continue to do what we're doing.

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Ten-Year Bone Cancer Survivor Rides 'Coast 2 Coast' - Curetoday.com

Cancer Stem Cell Market Size Research Study including Growth Factors, Types and Application to 2025 – Daily News Collectors

The Cancer Stem Cell Market Report provides key strategies followed by leading Cancer Stem Cell industry manufactures and Sections of Market like- product specifications, volume, production value, Feasibility Analysis, Classification based on types and end user application areas with geographic growth and upcoming advancement. The Cancer Stem Cell market report provides comprehensive outline of Invention, Industry Requirement, technology and production analysis considering major factors such as Revenue, investments and business growth.

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The well-established players in the market are:

Thermo Fisher Scientific, Inc.,AbbVie, Inc.,Merck KGaA,Bionomics,LONZA,Stemline Therapeutics, Inc,Miltenyi Biotec,PromoCell GmbH,MacroGenics, Inc.,ONCOMED PHARMACEUTICALS INC,Irvine Scientific,STEMCELL Technologies Inc,Sino Biological Inc,BIOTIME, INC.

This report for Cancer Stem Cell Market discovers diverse topics such as regional market scope, product market various applications, market size according to specific product, sales and revenue by region, manufacturing cost analysis, Industrial Chain, Market Effect Factors Analysis, market size forecast, and more.

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Product Type: Treatment Type,o Autologous Stem Cell Transplants,o Allogeneic Stem Cell Transplants,o Syngeneic Stem Cell Transplants,o Bone Marrow Stem Cell Transplants, Disease Type,o Breast Cancer,o Blood Cancer,o Lung Cancer,o Brain Cancer,o Colorectal Cancer,o Pancreatic Cancer,o Bladder Cancer,o Liver Cancer,o Others

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The report outlines the regulatory framework surrounding and governing numerous aspects of the market. At the end, Cancer Stem Cell industry development rival view, the industry scenario, samples, research conclusions are described. The important examination incorporated from 2014 to 2019 and till 2024 makes the report helpful assets for industry officials, promoting, sales, directors, experts, trade consultants, and others looking for key industry information with clearly given tables and charts.

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