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Global Stem Cell Therapy Market Analysis and Forecast, 2019-2029: Focus on Treatment Type, Cell Source, Indication,11 Countries’ Data, and Competitive…

DUBLIN, Sept. 20, 2019 /PRNewswire/ -- The "Global Stem Cell Therapy Market: Focus on Treatment Type, Cell Source, Indication,11 Countries' Data, and Competitive Landscape - Analysis and Forecast, 2019-2029" report has been added to ResearchAndMarkets.com's offering.

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Key Questions Answered in this Report:

The Global Stem Cell Therapy Industry Analysis projects the market to grow at a significant CAGR of 27.99% during the forecast period, 2019-2029.

The global stem cell therapy market growth has been primarily attributed to the major drivers in this market such as the increasing prevalence of chronic diseases, rising number of clinical trials for cell-based therapy, steady investment, and consolidation in the regenerative medicine market, and favorable regulatory environment.

The market is expected to grow at a significant growth rate due to the opportunities that lie within its domain, which include product approvals, declining product price, and increasing adoption rate.

However, there are significant challenges which are restraining the market growth. High treatment cost, the exorbitant cost required for set up, and ethical considerations related to the use of embryonic stem cells are the challenges faced by the market.

Key Topics Covered:

Executive Summary

1 Product Definition

2 Scope of the Work2.1 Overview: Report Scope2.2 Segmentation of the Global Stem Cell Therapy Market2.3 Assumptions and Limitations2.4 Key Questions Answered in the Report2.5 Base Year and Forecast Period

3 Research Methodology3.1 Overview: Report Methodology

4 Global Stem Cell Therapy Market4.1 Market Overview4.2 Introduction of Stem Cell Therapy4.3 Application of Stem Cells in Different Therapeutic Areas4.4 Market Dynamics4.5 Global Market Scenario4.6 Assumptions and Limitations

5 Competitive Landscape5.1 Overview5.2 Key Developments and Strategies5.2.1 Collaborations, and Partnerships5.2.2 Approvals and Clinical Studies5.2.3 Funding5.2.4 Business Expansions5.2.5 Product Launches and Developments5.2.6 Mergers and Acquisitions5.2.7 Others5.3 Market Share Analysis

6 Industry Insights6.1 Regulatory Scenario6.2 Regulatory Designations6.3 Expedited Designation Vs. Traditional Approval Timelines:6.4 Regulatory Challenges:

7 Global Stem Cell Therapy Market (by Treatment Type)7.1 Overview7.2 Key Trends of the Global Stem Cell Therapy Market (by Treatment Type)7.3 Autologous Treatment7.4 Allogenic Treatment

8 Global Stem Cell Therapy Market (by Cell Source)8.1 Overview8.2 Key Trends of the Global Stem Cell Therapy Market (by Cell Source)8.3 Bone Marrow and Peripheral Blood8.4 Adipose Tissue8.5 Placenta and Umbilical Cord8.6 Embryo8.7 Others

9 Global Stem Cell Therapy Market (by Indication)9.1 Overview9.2 Key Trends of the Global Stem Cell Therapy Market (by Indication)9.3 Orthopaedic and Dental9.4 Wounds and Injuries9.5 Cardiology and Neurology9.6 Immunology and Inflammatory9.7 Oncology and Metabolism9.8 Others

10 Global Stem Cell Therapy Market (by Region)10.1 Overview10.2 North America10.2.1 Overview10.2.2 U.S.10.2.3 Canada10.3 Europe10.3.1 Overview10.3.2 Germany10.3.3 U.K.10.3.4 France10.3.5 Italy10.3.6 Rest-of-Europe10.4 Asia-Pacific10.4.1 Overview10.4.2 Japan10.4.3 Australia10.4.4 China10.4.5 South Korea10.4.6 India10.4.7 Rest-of-Asia-Pacific10.5 Rest-of-the-World10.5.1 Overview10.5.2 Middle East and Africa10.5.3 Latin America

11 Company Profiles

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Global Stem Cell Therapy Market Analysis and Forecast, 2019-2029: Focus on Treatment Type, Cell Source, Indication,11 Countries' Data, and Competitive...

4 Stocks to Focus on Global Biotechnology Reagents Market – Yahoo Finance

Advancement in the field of biotechnology along with growing need of reagents in drug testing and stem cell research is pushing demand for biotechnology reagents worldwide. Therefore it would be prudent to take a look at a couple of stocks operating in the segment that are poised to gain from the trend.

Promising Growth for Biotechnology Reagents

The global biotechnology reagents market is poised for impressive growth, according to a reportby research firm Research and Markets. Increasing research on stem cells is a major factor pushing this industry. Per the research firm, the industry is set to witness a compound annual growth rate of about 9% during the forecast period 2019-2023.

Trends Driving Demand for Reagents

The biotechnology sector has been doing remarkably well this year, despite the lackluster performance by the broader healthcare sector. In fact, the SPDR S&P Biotech ETF (XBI) has outperformed the broader Health Care Select Sector SPDR Fund (XLV) on a year-to-date basis. The former has increased 15.2% while the latter has only moved 6.2% higher.

The increasing use of biotechnology reagents is a prominent reason behind the significant growth of the biotechnology sector. Reagents are a crucial element in the area of drug discovery and these have high demand in research, therapeutics and commercial applications.

This is why biotechnology reagents have attracted heavy investments in research and development lately and will continue to do so. In fact, reagents have a rather broad spectrum of application in stem cell research. These are used as biomarkers to visualize cell and tissue lines.

Stem cell research is growing by leaps and bounds, thanks to its many benefits that include treatment of an array of diseases. Therefore with the rise in stem cell research, demand for reagents is poised to rise as well.

Our Choices

We have therefore chosen four stocks that are revolutionizing the global biotechnology reagents market which investors could consider in their portfolio. All of these stocks carry a Zacks Rank #2 (Buy) or 3 (Hold).

Agilent Technologies, Inc. A is an analytical laboratory instrument manufacturing company. The companys Dako brand offers high-quality reagents, diagnostic antibodies, instruments and software solutions that assist in the treatment of cancer patients.

Agilent Technologies carries a Zacks Rank #2. The companys stock price has outperformed the Zacks Electronics - Testing Equipmentmarket on an annualized basis (+10.4% vs +0.6%). The Zacks Consensus Estimate for its current-year earnings has risen 1% over the past 60 days. You can seethe complete list of todays Zacks #1 Rank stocks here.

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Danaher Corporation DHR is a conglomerate and manufacturer of medical products. The companys life sciences wing offers sophisticated equipment for biological research.

Danaher carries a Zacks Rank #2. The companys stock price has already outperformed the Zacks Diversified Operationsmarket on a year-to-date basis (+34.9% vs -5.2%). The Zacks Consensus Estimate for its current-year earnings has risen 0.2% over the past 60 days.

Thermo Fisher Scientific Inc.TMO is a manufacturer of analytical instruments, equipment and reagents.

Thermo Fisher Scientific carries a Zacks Rank #3. The companys stock price has outperformed the Zacks Medical - Instrumentsmarket on a year-to-date basis (+21.3% vs -5.7%). The Zacks Consensus Estimate for its current-year earnings has risen 0.4% over the past 60 days.

General Electric Companys GE life sciences wing operates in the cell and gene therapy industry. The company offers end-to-end solutions that include software and equipment that drive uniformity to dependable reagents.

General Electric carries a Zacks Rank #3. The stock price has outperformed the Zacks Diversified Operationsmarket on a year-to-date basis (+24.5% vs +20.3%). The Zacks Consensus Estimate for its current-year earnings has risen 1.6% over the past 60 days.

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Experts extracted 7 stocks from the list of 220 Zacks Rank #1 Strong Buys that has beaten the market more than 2X over with a stunning average gain of +24.6% per year.

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Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free reportDanaher Corporation (DHR) : Free Stock Analysis ReportGeneral Electric Company (GE) : Free Stock Analysis ReportThermo Fisher Scientific Inc. (TMO) : Free Stock Analysis ReportAgilent Technologies, Inc. (A) : Free Stock Analysis ReportTo read this article on Zacks.com click here.Zacks Investment Research

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4 Stocks to Focus on Global Biotechnology Reagents Market - Yahoo Finance

Editing Muscle Stem Cells with CRISPR Treats Mouse Model of Muscular Dystrophy – DocWire News

A research team from the University of Missouri School of Medicine has recently used CRISPR to edit a genetic mutation that contributes to Duchenne muscular dystrophy (DMD). This rare and debilitating genetic disorder is characterized by loss of muscle mass and physical impairment. By using this powerful gene-editing technology, these MU School of Medicine researchers have successfully treated mouse models of the disease. This work was published this summer in the journal Molecular Therapy.

Those with DMD possess a specific mutation that hinders the production of the dystrophin protein, which contributes to the structural integrity of muscle tissue. In the absence of this protein, the muscle cells weaken and eventually die. Pediatric patients with the condition often lose their ability to walk and can even lose the function of muscles that are essential for respiration and heart contractions.

Research has shown that CRISPR can be used to edit out the mutation that causes the early death of muscle cells in an animal model, explained senior author Dongsheng Duan, PhD, Margaret Proctor Mulligan Professor in Medical Research in the Department of Molecular Microbiology and Immunology at the MU School of Medicine. However, there is a major concern of relapse because these gene-edited muscle cells wear out over time. If we can correct the mutation in muscle stem cells, then cells regenerated from the edited stem cells will no longer carry the mutation. A one-time treatment of the muscle stem cells with CRISPR could result in continuous dystrophin expression in regenerated muscle cells.

Working alongside other researchers from MU, the National Center for Advancing Translational Sciences, Johns Hopkins School of Medicine and Duke University, Duan aimed to genetically modify muscle stem cells in mice. These scientists first edited the gene using an adeno-associated virus known as AAV9. Being this specific viral strain was recently approved by the FDA in treating spinal muscular atrophy, the researchers saw it as a viable candidate in treating DMD.

We transplanted AAV9 treated muscle into an immune-deficient mouse, said lead author Michael Nance, an MD-PhD program student in Duans lab. The transplanted muscle died first then regenerated from its stem cells. If the stem cells were successfully edited, the regenerated muscle cells should also carry the edited gene.

Upon analyzing the regenerated muscle tissue, the researchers found that its cells contained the edited gene, supporting their reasoning. The team then tested whether the muscle stem cells in mice with DMD could be genetically edited using CRISPR. These findings also supported their hypothesis, with the stem cells in the diseased tissue sustaining these edits and the regenerated cells successfully producing dystrophin.

This finding suggests that CRISPR gene editing may provide a method for lifelong correction of the genetic mutation in DMD and potentially other muscle diseases, explained Duan. Our research shows that CRISPR can be used to effectively edit the stem cells responsible for muscle regeneration. The ability to treat the stem cells that are responsible for maintaining muscle growth may pave the way for a one-time treatment that can provide a source of gene-edited cells throughout a patients life.

Duan and colleagues hope that future research will help this stem cell CRISPR therapy become a revolutionary treatment for children with DMD.

Link:
Editing Muscle Stem Cells with CRISPR Treats Mouse Model of Muscular Dystrophy - DocWire News

Testing chemicals for birth defects using stem cells, not mice – UC Riverside

Researchers at the University of California, Riverside, are part of an ambitious plan at the U.S. Environmental Protection Agency, or EPA, to eliminate animal testing by 2035. Their contribution: developing a way to test whether chemicals cause musculoskeletal birth defects using lab-grown human tissue, not live animals.

Nicole zur Nieden, an associate professor of molecular, cell, and systems biology, and David Volz, an associate professor of environmental toxicology, are both experts on alternatives to regulatory toxicity testing and chemicals policy and regulation. They received $849,811 to grow human stem cells into bone-like tissue to test industrial and environmental chemicals that might interfere with fetal growth.

Birth defects that affect musculoskeletal tissues can be caused by chemical ingredients in pesticides, fungicides, paints, and food additives. Harmful chemicals must be identified through testing in order to be regulated. Currently, this testing is done on live animals, usually rodents such as mice.

The UC Riverside project, led by zur Nieden, will stimulate human pluripotent stem cells, which have the capacity to develop into any sort of cell, with agents that direct them to form bone cells. The cells will pass through the same developmental stages and be subject to the same molecular cues as in a human embryo. The researchers will expose the cells to selected chemicals at critical junctures, then assess them using advanced imaging and next-generation sequencing techniques.

Bone cells can develop through three different pathways. zur Nieden will use chemicals known to affect specific routes of bone development to look for patterns in how the chemicals affect these origins. The patterns will serve as blueprints for testing unknown chemicals. Next, the researchers will test unknown chemicals and compare them to previously compiled libraries of compounds that have already been tested in animals to see how accurate the petri dish, or in vitro, tests are for assessing risk.

A hallmark feature of bone-forming cells is that they make a bony matrix out of little crystals called hydroxyapatite, which eventually form calcium phosphate, the white stuff on the surface of all bones. Cost-saving visual analysis can help identify defects in calcium.

Calcium crystals appear white when viewed with your eyes, said zur Nieden. But when you view the cultures using phase contrast microscopy, it inverts the light so the normal crystals appear black. Abnormal crystals will have more white and shades of gray. You can use an image analysis algorithm to measure the blackness in images to determine if the calcium has formed correctly or not.

Scientists have known for a long time that animals differ from humans in important developmental and physiological ways, and that animal test results are not always reliable for people. Moreover, animal research is expensive and time-consuming, as well as increasingly untenable for ethical reasons. Non-animal alternatives have been in development for nearly 25 years, and some are already standard.

To the general public, the EPAs announcement seemed to come out of nowhere, said Volz, whose lab will sequence messenger RNA in chemical-exposed bone cells from zur Niedens lab to look for changes in gene expression. It didnt happen overnight. That train has already left the station.

Volz said the EPAs Science to Achieve Results Program, through which UC Riverside received the new grant, has been funding research on animal alternatives for more than 10 years.

The EPAs plan to end animal testing by 2035 follows up on earlier changes to the Toxic Substances Control Act, or TSCA, enacted in 1976. TSCA authorizes the EPA to regulate chemicals found in consumer products such as cleaning agents, furniture, paint, carpeting, clothing, and other consumer goods. Regulation under TSCA does not apply to chemicals in food, drugs, cosmetics, and pesticides, which are regulated under different laws.

Even after TSCA, thousands of common chemicals used in everything from plastic to sunscreen have never been tested for safety in humans. In 2016, Congress passed the Lautenberg Chemical Safety Act, amending TSCA to close the loophole for industrial chemicals. The law mandated the EPA to evaluate existing chemicals with clear and enforceable deadlines, and to develop risk-based chemical assessments. It promoted the use of non-animal testing methods, a move sought by both industry and animal rights groups.

The new EPA plan introduces an aggressive timeline for ramping up development of non-animal tests that can accurately predict toxicity in humans. Volz said the United States lags behind some other countries around the world, which have already greatly reduced animal testing. He said he interacts with fewer and fewer students interested in research involving animal experiments, and that our culture is shifting toward a desire to reduce animal suffering.

But neither Volz nor zur Nieden are sure animal testing can ever be replaced completely, a position echoed by the EPA memo, which states that after 2035, animal tests will be approved on a case-by-case basis. Some chemicals, for example, are not directly toxic to cells but become toxic after they are metabolized in the body.

If your result is that the chemical does not interfere with a human stem cell developing in a dish, how sure can you be thats not really happening in humans? The best way we have to assess that is an animal experiment, zur Nieden said. At the same time, we want to do this in an appropriate way. We need to think about, is this really necessary? Can we look at the question some other way?

zur Nieden thinks we need a tiered system, with in vitro tests weeding out the most toxic chemicals first, and animal tests used where in vitro tests dont reveal toxicity.

If you cannot fully replace an animal test with an in vitro method, you can at least decrease suffering of the animal. If you think about a highly toxic chemical that has effects on the mom as she is exposed during pregnancy as well as on the developing embryos, if you can use an in vitro test system to find all these strong toxic chemicals, you will not need to test them in an animal, she said.

Previous versions of the test system zur Nieden will use for the new musculoskeletal research have been able to identify embryotoxic chemicals for other tissues, such as heart tissue, with almost 100% accuracy.

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Testing chemicals for birth defects using stem cells, not mice - UC Riverside

Mutant Tau Stiffens Axon Cytoskeleton Near Soma – Alzforum

20 Sep 2019

Changes to the microtubule-binding protein tau cause it to fall off these struts and aggregate, forming neurofibrillary tangles. But is that the reason for tau toxicity? A study in the September 18 Neuron suggests that V337M tau, a variant associated with frontotemporal dementia, causes morphological changes at the base of the axon. Scientists led by Li Gan, then at the Gladstone Institute of Neurological Disease, San Francisco, found that neurons derived from patients with the mutation are hyperexcitable. They had unusually short, unresponsive axon initial segments (AIS), which usually initiate action potentials and steer neuronal plasticity. Gans results suggest that this FTD mutation robs neurons of a mechanism for maintaining electrical homeostasis.

The initiation of axon potential firing has received little attention in neurodegeneration research, said Jrgen Gtz, What I really like about this paper is the use of complementary techniques to mechanistically dissect the effect of FTD mutant tau on neuronal function.

Because many FTD-causing tau mutations occur in the microtubule-binding domain, scientists believed they lead to disease by weakening microtubules (Rossi and Tagliavini, 2015; Hong et al., 1998). However, studies have mostly found that removing tau leaves microtubules intact (Roberson et al., 2007). On the other hand, FTD patients have hyperexcitable neurons, seizures, and highly synchronized neuronal networks (Beagle et al., 2017). Could the mutant protein alter neuronal excitability in some way?

Plasticity Deficit. In iPSC-derived wild-type neurons (left) the axon initial segment (green) is long initially (top)and shrinks after chronic depolarization with KCl (bottom). In neurons with V337M tau (right), the AIS starts out and remains short. [Courtesy of Sohn et al., 2019.]

To find out, first author Peter Dongmin Sohn and colleagues focused on the AIS. The closest part of the axon to the soma, the AIS contains a high concentration of voltage-gated ion channels and triggers action potentials. It regulates neuronal excitability by lengthening or shrinking in response to less or more activity, respectively. This remodeling relies on a reorganization of the cytoskeleton, in particular ankyrin G. Staining for AnkG revealed that the AIS shrank, making it less excitable, when Sohn and colleagues depolarized wild-type neurons for two days (see image at left). In contrast, in iPSC-derived neurons from a patient with the V337M tau mutation, the AIS was about 20 percent shorter to begin with, and chronic depolarization did not change its length. This suggested the region was less plastic. If the researchers used CRISPR-Cas9 to correct the mutation, then the initial length of the AIS and its plasticity matched that of wild-type cells.

The researchers next compared electrophysiological properties of mutant and control neurons. In culture, neurons carrying mutant tau more often fired in synch, having longer network bursts containing more spikes, than did wild-type neurons. This suggested the mutated tau caused a type of hyper-synchrony. After a two-day depolarization, these neurons fired six times faster, while the rate stayed steady in isogenic controls.

To find out how the mutant tau might be interfering with homeostatic control of neuronal excitability, the authors examined tau binding partners in the AIS. Tau interacts with end-binding protein 3 (EB3), another component of the AIS cytoskeleton (Sayas et al., 2015). EB3 stabilizes the AIS by linking microtubules to AnkG.

Getting a Grip. At left, wild-type tau (blue) binds EB3 (green), which anchors AnkG (yellow) to microtubules (white). To the right, mutant tau (red) binds more tightly to EB3 and clamps it in place, making the AIS rigid and unchanging. [Courtesy of Sohn et al., 2019.]

Sohn and colleagues determined that tau binds EB3 directly, and that V337M tau does so more tightly. In tau V337M neurons, EB3 levels in the AIS were 40 percent higher than in wild-type. Whats more, rather than distributing throughout the AIS cytosol, EB3 gathered just under the plasma membrane, corralling AnkG. In all, the data suggested that through its grip on EB3, tau concentrated the protein in the AIS and immobilized AnkG such that it couldnt respond to electrical activity.

Would removing EB3 rescue plasticity in V337M neurons? Suppressing translation with siRNA, Sohn reduced levels by 80 percent, which restored both the length and plasticity of the AIS. Reducing mutant tau in these neurons by 40 percent had similar effectsEB3 levels shrank in the AIS, and plasticity was restored.

Our study provides a completely different explanation for why tau is toxicnot because it forms aggregates, but because it binds to cytoskeletal proteins important for plasticity and makes rigid structures, Gan, who has since moved to Weill Cornell Medicine, New York, told Alzforum. This is an unexpected aspect of tau pathology. Gan plans to test whether this is true in other FTD mutations, and wonders about tau-induced hyperexcitability in Alzheimers disease, in which tau is not mutated.

This study provides an important demonstration of the utility of human iPSC models to reveal changes in neuroplasticity that may lead to disruption of brain circuitry over the course of disease, wrote Stephen Haggarty, Massachusetts General Hospital, Boston, to Alzforum. [It] provides further validation of therapeutic strategies seeking to reduce expression of pathological forms of tau.

Not all tau mutations work in this manner. In the September 19 Stem Cell Reports, researchers led by Hideyuki Okano of Keio University, Tokyo, report a different effect of the R406W. This mutation lies outside the microtubule-binding domain, but has nevertheless been reported to impede tau binding to those intracellular rails. Also using patient-derived neurons, first author Mari Nakamura and colleagues found that R406W tau mislocalizes to dendrites, as was seen before in cell and animal overexpression models (Thies and Mandelkow, 2007; Jan 2011 news). There it disrupts mitochondrial transport and causes axonal degeneration.

In neither study do the cell models rely on overexpression of the mutant tau protein, thereby overcoming issues often inherent to artificially engineered systems, Haggarty wrote.Gwyneth Dickey Zakaib

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Mutant Tau Stiffens Axon Cytoskeleton Near Soma - Alzforum

Global Cell Harvesting Market is Booming Market to Rapidly Growing $ 387.9 Million by 2026 with Top Key Players PerkinElmer Inc, Bertin, Tomtec,…

The Cell Harvesting Market research report provides the generic overview of the market manufactures, things and Product application scope. This report considers with various pieces of the professional the market size, status, industry examples and conjecture, the report in like manner gives brief information of the contenders and the specific improvement openings with key drivers. It provides the market segmentation analysis including the qualitative and quantitative research methodology incorporating the impact of economic and non-economic perspective.

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This market report defines the market trends and forecast the upcoming opportunities and threats of the cell harvesting market in the next 8 years. Cell harvesting is extracting the cells either from bone marrow and peripheral blood cells and culturing the cells in the culture dish containing nutrient media. Cell harvesting is used in the cell therapy as well as in gene therapy. University of California developed a cure for bubble baby disease for new born babies by using the cell harvesting in stem cells and gene therapy.

The Cell Harvesting research report thoroughly explains the primary and secondary research techniques to arrive at interferences. It has been aggregated on the basis of the several dynamic aspects of the businesses. Market Dynamics Like Market Drivers, Restraints and Threats are also encompassed in the report.

The Emerging Key Market players of the Market are included in the Cell Harvesting research report. The key competitors are also analyzed in the report. the market report is considered to be a combination of the current trends.

Some of the major players operating in the global cell harvesting market are PerkinElmer Inc, Bertin, Tomtec, Terumo BCT, HynoDent AG, Avita Medical, Argos Technologies, SP Industries, Teleflex Incorporated, Arthrex, Inc, Thomas Scientific, Brand GMBH, Brandel, Cox Scientific, Connectorate, Scinomix, Adstec.

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Global Cell Harvesting Market, By Type (Manual Cell Harvesters and Automated Cell Harvesters), By Application (Biopharmaceutical Application, Stem Cell Research and other Applications), By End Users (Hospitals, Ambulatory Centers, Clinics, Community Healthcare, Others), By Geography (North America, South America, Europe, Asia-Pacific, Middle East and Africa) Industry Trends and Forecast to 2025

The market is geographically Divided into various regions such as

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Global Cell Harvesting Market is Booming Market to Rapidly Growing $ 387.9 Million by 2026 with Top Key Players PerkinElmer Inc, Bertin, Tomtec,...

Stem Cells May Be Able To Repair The Brain – Anti Aging News

Transplanted brain stems cells have survived without using anti-rejection drugs in mice by exploiting a feature of the immune system, findings may open new paths for stem cell transplants to help repair the brain.

Johns Hopkins Medicine research may have developed a way to transplant certain protective brain cells without the need for lifelong anti-rejection drugs, as published in the journal Brain; the approach selectively circumvents responses from the immune system against foreign cells to allow the transplanted cells to survive and thrive to protect brain tissues long after stopping immunosuppressive drugs.

"Because these conditions are initiated by a mutation causing dysfunction in one type of cell, they present a good target for cell therapies, which involve transplanting healthy cells or cells engineered to not have a condition to take over for the diseased, damaged or missing cells," says Piotr Walczak, M.D., Ph.D., associate professor of radiology and radiological science at the Johns Hopkins University School of Medicine.

The researchers are working on developing ways to stop the immune system responses without side effects, and investigated ways to manipulate T cells which are the immune systems infection fighting force that attack foreign invaders, specifically focussing on a series of costimulatory signals that T cells must encounter in order to begin attack.

"These signals are in place to help ensure these immune system cells do not go rogue, attacking the body's own healthy tissues," says Gerald Brandacher, M.D., professor of plastic and reconstructive surgery and scientific director of the Vascularized Composite Allotransplantation Research Laboratory at the Johns Hopkins University School of Medicine and co-author of this study.

To exploit natural tendencies of these costimulatory signals that train the immune system to accept the transplanted cells as being part of self the researchers used CTLA4-lg and anti-CD154 antibodies to keep the T cells from beginning an attack by blocking signals.

Protective glial cells that produce myelin sheath that surrounds neurons that were genetically engineered to glow were injected into mice brains so they could be studied. Glial cells were transplanted into three types of mice: those engineered to not for glial cells that create myelin sheath; normal mice; and those engineered not to be able to mount an immune response.

Antibodies were used to block an immune response, stopping treatment after six days, and specialized cameras were used to detect the glowing cells and pictures of the mice brains to look for the presence or absence of the transplanted cells. Cells transplanted into control mice without antibody treatment began to die off immediately and was no longer detected after 21 days; while those receiving antibody treatment maintained significant levels of transplanted cells for over 203 days even in the absence of treatment.

"The fact that any glow remained showed us that cells had survived transplantation, even long after stopping the treatment," says Shen Li, M.D., lead author of the study. "We interpret this result as a success in selectively blocking the immune system's T cells from killing the transplanted cells."

To see whether the transplanted glial cells survived well enough to create the myelin sheath key structural differences were looked at between mice brains with and without thriving glial cells using MRI imaging; cells in the treated animals were found to be populating the appropriate parts of the brain. Results confirm the transplanted cells were able to thrive and assume normal function to protect neurons.

Although results are preliminary the cells were delivered to thrive in a localized portion of the mouse brain. Researchers hope to combine findings with studies on cell delivery methods to the brain in the future to help repair the brain more globally.

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Stem Cells May Be Able To Repair The Brain - Anti Aging News

MS News that Caught My Eye: Stem Cell Transplants, Remyelination Agent, Tecfidera Study, Plasma Exchange and Tysabri-linked PML – Multiple Sclerosis…

Its been a big week for interesting stories, as the annual meeting of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) has just concluded. The conference offered much to engage healthcare professionals and researchers, but the following are some presentations that appealed to me as a multiple sclerosis (MS) patient.

The debate continued over the risks versus the rewards of stem cell transplants. Joachim Burman, MD, PhD, a researcher at Uppsala University in Sweden, made the case as to why autologous hematopoietic stem cell transplantation may be the most effective MS treatment so far, and why it should be made widely available. A single administration can yield prolonged benefits, and most patients achieve no evidence of disease activity status for at least five years. But safety concerns exist, and the lack of control groups tempers the positive results in studies.

Stem cell therapy, or stem cell transplant, is an emerging yet controversial treatment approach for multiple sclerosis (MS). While some data uphold it as one of the most efficacious MS treatments, to date there have been no controlled studies comparing it to conventional medicines and providing more robust evidence regarding its safety and clinical benefit.

Under the topic HSCT and stem cell treatment in MS,a group of researchers discussed the promise and current challenges of stem cell transplant at the 35thCongress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), being held Sept. 1113 in Stockholm.

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As those of us who have multiple sclerosis know, finding a way to repair the damage to the myelin that covers our nerves would be like discovering the Holy Grail. This research, which looked into whether a potential remyelination agent is safe and might provide some repair, gives some hope.

Treatment with a potential remyelinating agent called liothyronine was safe and well-tolerated by people with multiple sclerosis (MS) in a Phase 1b trial. Preliminary results also suggested benefits in cognition, motor function, and fatigue.

The study, A Phase 1b, open-label study to evaluate the safety and tolerability of the putative remyelinating agent, liothyronine, in individuals with multiple sclerosis, was presented today at the 35thCongress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), by Scott D. Newsome, professor of neurology at Johns Hopkins University School of Medicine.

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Out of 618 RRMS patients followed for 10 years, 192 of them were on continuous Tecfidera (dimethyl fumarate) twice daily treatment throughout that time, and many reported that the disease-modifying therapy (DMT) had helped them. Just over half of these patients remained relapse-free, and 64 percent had no signs of confirmed disability progression over that period. In a separate presentation, researchers found that the DMTs Tysabri (natalizumab) and Lemtrada (alemtuzumab) were better at reducing the annualized relapse rate than Tecfidera.

New 10-year data from the Phase 3 ENDORSE trial confirms the long-term benefits of Biogens Tecfiderafor patients with relapsing-remitting multiple sclerosis(RRMS), the most common form of this disease.

Real-world data from another study also showed Tecfidera to be superior to several other disease-modifying therapies for relapsing MS, namely Copaxone (glatiramer acetate), Aubagio(teriflunomide), andinterferon beta.

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Treatment with Tysabri (natalizumab) carries the risk of triggering the often fatal brain infection progressive multifocal leukoencephalopathy (PML). Several treatments have been tried to attack PML, one of which is a type of blood cleansing called PLEX. It was hoped that PLEX would quickly clear Tysabri from the blood, and in doing so, would improve PML survival rates. Unfortunately, this study reports that two years after PML was detected the survival rate of patients treated with PLEX was no different than patients who had not received the treatment.

Use of plasma exchange (PLEX) is not effective for treating progressive multifocal leukoencephalopathy (PML), a dangerous brain infection that has been associated with using the multiple sclerosis (MS) medicine Tysabri (natalizumab), a real-world study contends.

The findings highlight the importance of closely monitoring Tysabri users to detect PML as early as possible.

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Note: Multiple Sclerosis News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Multiple Sclerosis News Today or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to multiple sclerosis.

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MS News that Caught My Eye: Stem Cell Transplants, Remyelination Agent, Tecfidera Study, Plasma Exchange and Tysabri-linked PML - Multiple Sclerosis...

Tiny ‘envelopes’ show promise for sun-damaged skin repair – Futurity: Research News

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Exosomes harvested from human skin cells are more effective at repairing sun-damaged skin cells in mice than popular retinol or stem cell-based treatments currently in use, according to a new proof-of-concept study.

Additionally, needle-free injections can deliver the nanometer-sized exosomes to the target cells.

Exosomes are tiny sacs (30150 nanometers across) that cells excrete and take up. They can transfer DNA, RNA, or proteins from cell to cell, affecting the function of the recipient cell. In the regenerative medicine field, researchers are testing exosomes as carriers of stem cell-based treatments for diseases ranging from heart disease to respiratory disorders.

Think of an exosome as an envelope with instructions insidelike one cell mailing a letter to another cell and telling it what to do, says corresponding author Ke Cheng, professor of molecular biomedical sciences at North Carolina State University and professor in the universitys joint biomedical engineering department with the University of North Carolina at Chapel Hill. In this case, the envelope contains microRNA, non-coding RNA that instructs the recipient cell to produce more collagen.

To test whether exosomes could be effective for sun-damaged skin repair, the researchers first grew and harvested exosomes from skin cells. They used commercially available human dermal fibroblast cells, expanding them in a suspension culture that allowed the cells to adhere to one another, forming spheroids. The spheroids then excreted exosomes into the media.

These 3D structures generate more procollagenmore potent exosomesthan you get with 2D cell expansion, says Cheng.

In a mouse model, Cheng tested the 3D spheroid-grown exosomes against three other treatments: retinoid cream; 2D-grown exosomes; and bone marrow derived mesenchymal stem cells (MSCs) exosomes, a popular stem cell-based anti-aging treatment currently in use.

The team compared improvements in skin thickness and collagen production after treatment. They found that skin thickness in 3D exosome treated mice was 20% better than in the untreated and 5% better than in the MSC-treated mouse. Additionally, they found 30% more collagen production in skin treated with the 3D exosomes than in the MSC treated skin, which was the second most effective treatment.

I think this study shows the potential for 3D exosomes to be used in anti-aging skin treatments, says Cheng. There are two major benefits to exosome treatments over conventional treatments: one, you can use donor skin cells from anyone to grow and harvest these exosomesthey arent cells, so you dont run the risk of rejection. And two, the treatment can be administered without needlesexosomes are small enough to be able to penetrate the skin via pressure, or jet injection methods.

Our hope is that eventually people may be able to bank skin samples and come back to them, or use donor exosome treatments that they can administer themselves. We believe that this work is an important step toward potentiating future human clinical trials in the prevention and treatment of cutaneous aging.

The work appears in ACS Nano.

The National Institutes of Health and the American Heart Association supported the research, in part.

Source: Tracey Peake for NC State

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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