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Back from Mexico, Indian Land teen hopes she gave herself the gift of life – The Herald


The Herald
Back from Mexico, Indian Land teen hopes she gave herself the gift of life
The Herald
Later this month, Grace will go Houston to see Dr. Ian Butler, a pediatric neurologist who's been treating her since late last year, and Dr. Stanley Jones. They'll evaluate her condition since the first stem cell treatment, and hopefully prescribe ...

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Back from Mexico, Indian Land teen hopes she gave herself the gift of life - The Herald

In a first, Mumbai doctors use dad’s cells to fight blood disorder – Times of India

MUMBAI: Three-year-old Kinaya Shah was diagnosed with thalassemia at the tender age of three months and has been undergoing regular blood transfusions ever since. The only cure for thalassemia is a bone marrow transplant (BMT), a form of stem cell therapy. Typically, the donor of the stem cells would be a sibling of the patient such that the stem cells of the donor are a near perfect match to those of the patient. The only complication was that Kinaya was a lone child.

So, city doctors in a first used stem cells donated by Kinaya's father - who was only a half or haploidentical match - to cure the child of the blood disorder. "We went to Vellore, Bangalore and Pune but no one was willing to do the transplant without a full match donor," said Kinaya's parents, Aneri and Shripal Shah. They approached Dr Santanu Sen at the Kokilaben Dhirubhai Ambani Hospital, Andheri, in October of 2016, after reading about a similar surgery that he had performed.

While haploidentical bone marrow transplants are carried out to cure leukaemia, it has only been done about half a dozen times for thalassemia in a couple of Indian cities. ``Haploidentical transplants are gradually increasing because of better techniques,'' said Dr Sen.

Dr Sen has completed 36 BMTs in the last two years, of which 12 were haploidentical donors. ``But this is the first time that a haploidentical transplant has been done in western India to cure thalassemia,'' he said.

Chennai-based haematologist Dr Revathy Raja said that there is a 85% chance of cure in thalassemia with a fully matched donor. ``The success rate falls to 70% with a half-match or haploidentical donor. We have hence not started it at our Chennai centre. Hopefully, techniques will further improve in the coming years,'' she said.

In order to perform the surgery, Dr Sen conditioned Kinaya's immune system over three months, with slight chemotherapy, to increase the chances of her body accepting the graft. "We found that her father's stem cells were a 70% match through genetic tests and decided to use them for the transplant. In the case that the graft was rejected we froze a couple of Kinaya's stem cells as insurance. The positive is that children have lower rejection rates for foreign cells as they have barely developed any active immunity," said Dr Sen. "BMT is the most viable treatment to cure thalassemia, the only barrier thus far was the necessity of a full match donor," he added.

However, Vinay Shetty of NGO Think Foundation, which works for thalassemia patients, said that it would be prudent to wait for a statistically significant number of successful halploidentical transplants before recommending it to all patients.

Post the three months of conditioning, stem cells were collected from her father's bone marrow and the transplant was performed on May 10, 2017. After several tests to confirm that the graft was accepted, Kinaya was finally discharged from the hospital on June 13.

"The future of thalassemia treatment probably lies in gene therapy, but at the moment, haploidentical transplants have made BMT much more accessible," said Dr Sen, adding that he has two more cases such as Kinaya lined up. Kinaya is expected to be completely independent of medication and any trace of thalassemia in the coming six months.

What is Thalassemia?

Thalassemia is a genetic blood disorder when the body produces abnormal hemoglobin. Patients require regular blood transplant and well as dietary control to ensure that blood irons level stay suppressed.

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In a first, Mumbai doctors use dad's cells to fight blood disorder - Times of India

Stem Cell Therapy for Glaucoma – Are We There Yet? – Newswise (press release)

Newswise There is great interest among glaucoma patients, scientists and doctors alike, in discovering regenerative therapies for the optic nerve and translating them from the laboratory to the clinic and stem cell therapy is one of several promising approaches being studied.

Recently, we heard from a glaucoma patient who enrolled in a patient-funded trial in which the person paid $20,000 and received stem cell injections around one eye. Patient-funded trials are studies in which patients pay to participate. This approach was developed because clinical trials are expensive and funding from traditional sources (such as the NIH, pharmaceutical companies, or private foundations) is decreasing.

Although patient-funded research would appear superficially to provide an avenue for patients to obtain therapies under investigation, it is controversial for a number of reasons, both scientific and ethical. Scientific considerations are deeply concerning: the gold standard for evaluating an experimental treatment is a randomized clinical trial in which participants are randomly allocated to the experimental treatment group or a control group that may not receive any treatment (placebo-controlled study), or may receive standard, approved therapies. Usually, study patients and doctors are both unaware of who receives which treatment and this study design minimizes bias towards a particular treatment.

In contrast, patient-funded trials do not have a control group, since it is extremely unlikely that patients would pay when there is a possibility of not receiving the experimental treatment. A control group is very important to determine if an experimental treatment really has an effect and also to compare the efficacy and risks of the experimental treatment versus other treatments. Although it is common in an early, phase 1 trial to treat perhaps the first 3-12 patients in an open-label, non-randomized study, by phase 2 trials, a randomized, masked design is ideal.

Even more concerning are the ethical considerations. These include disparity in access to the treatment, and risk of exploitation of vulnerable patients who have exhausted all treatment options and may be willing to undergo an unproven treatment at any cost. In addition, a reported lack of proper oversight and monitoring for patient-funded trials implies the possibility that these may be thinly-veiled attempts to make money by the treating clinic or physician before proper FDA approval of new treatment methods.

Since a gold standard research study is not always feasible, decisions regarding the benefits and risks of a particular treatment may have to be made with the evidence at hand. Given these considerations, we consulted Dr. Jeffrey Goldberg regarding stem cell therapy for glaucoma. Dr. Goldberg is a leading expert in therapies to regenerate the optic nerve and is part of the Catalyst for Cure team. His laboratory is developing novel stem cell approaches for glaucoma, and working steadily towards a translational program to bring discoveries out of the laboratory and into human testing once safety and efficacy in pre-clinical models is established.

Q: Dr. Goldberg, how might stem cells be helpful for patients with glaucoma?

A: Stem cells may be helpful for patients with glaucoma in different ways. Stem cells can be turned into trabecular meshwork cells in the front of the eye and transplanted in such a way as to lower eye pressure. This is an interesting approach but is not fundamentally about vision restoration.

For protecting or restoring vision, we really need to talk about stem cells in the back of the eye, at the retina. There, stem cells may have two positive effects. First, early in the disease, they may protect retinal ganglion cells from degenerating providing a neuroprotective effect. Later in the disease when patients have lost considerable numbers of retinal ganglion cells and optic nerve axons, and have thereby lost considerable vision, stem cells may be useful to replace lost ganglion cells and restore the connections from the eye to the brain. This last approachregrowing optic nerve fibers back to the brainhas been the most challenging but its also the most exciting.

What is the current status of research on stem cell therapy for glaucoma?

Our laboratory and a number of other laboratories have made considerable progress on the two main fronts of bringing stem cell therapy to optic nerve restoration for glaucoma. First, we and others have discovered molecular pathways that can be used to coax stem cells to turn into neurons that look and act like real retinal ganglion cells. This will allow us to turn large numbers of stem cells into retinal ganglion cells for cell replacement therapy. Second, we are just beginning to make progress in transplanting retinal ganglion cells into the retina in pre-clinical models, to study their integration into the adult retina, how they respond to light and grow back down the optic nerve to the brain. Together these advances have brought us to an exciting moment in stem cell research for optic nerve restoration in glaucoma.

Are you aware of any studies in which stem cell therapy stabilized or reversed vision loss from glaucoma?

Stem cells have not yet been properly tested in patients with glaucoma to look for their ability to stabilize or reverse vision loss. The careful move from the laboratory to clinical testing is still ahead of us, although with the intellectual energy and resources ready to deploy, such proper testing may not be far off.

If your family member had vision loss from glaucoma, would you recommend stem cell therapy at this time?

I am often asked by my patients if they should sign up for a patient-funded trial for stem cells for glaucoma, and I am in the habit of counseling against this. I am not aware of any properly designed stem cell trials for glaucoma with well-tested cell therapies being moved to human testing at this time, but I do think these will come.

Are there any risks or complications reported with stem cell therapy for glaucoma?

Indeed the risks for undergoing stem cell injections in any trial could be significant. Risks of infection, inflammation, and more severe vision loss will always be present; we are publishing a paper about 3 patients in the U.S. who participated in a patient-funded trial and lost significant vision due to severe inflammation in their eyes called endophthalmitis. These 3 unfortunate patients point to the importance of a cell therapy first undergoing proper testing in pre-clinical models before moving to human testing. Then, with properly designed and sequenced trials, I believe cell therapies can be safely tested in the eye as with the rest of the body. Indeed there are a number of cell therapies for macular degeneration already in human testing with a reassuring safety record thus far.

What type of study would you design for evaluating stem cell therapy with glaucoma?

After demonstrating safety and efficacy of a cell therapy product in pre-clinical models, a small pilot study designed to assess for safety after injection in humans and analysis of the results should be the first step. After this, a move to a randomized trial with a control group and masked observers will be best to assess efficacy in phase 2 and eventually phase 3 trials.

Article by Sunita Radhakrishnan, MD, Jeffrey L. Goldberg, MD, PhD, and Andrew Iwach, MD.

For additional information on this topic, following are links to related articles from the American Academy of Ophthalmology:

Unregulated Stem Cell Treatments Can Be Dangerous [June 6, 2017]

American Academy of Ophthalmology Statement on Stem-Cell Therapy for Treating Eye Disease [March 20, 2017]

Unapproved stem cell treatment blinds 3 patients [March 17, 2017]

Stem Cell Therapy for Eye Disease: What You Need to Know [June 24, 2016]

Intraocular Stem Cell Therapy [June 2016]

Excerpt from:
Stem Cell Therapy for Glaucoma - Are We There Yet? - Newswise (press release)

On the cusp of payoffs for patients, stem cell therapy faces threat from unregulated clinics – STAT

TV documentary on pain treatment funded by doctor with

TV documentary on pain treatment funded by doctor with industry ties

For some chronic pain patients, without opioids, life would

For some chronic pain patients, without opioids, life would be torture

Googles bold bid to transform medicine hits turbulence under

Googles bold bid to transform medicine hits turbulence under a divisive CEO

At first meeting of Trumps opioid commission, health advocates

At first meeting of Trumps opioid commission, health advocates plead for Medicaid spending

This bill would reinstate a controversial drug discount for

This bill would reinstate a controversial drug discount for some hospitals

Up and down the ladder: The latest comings and

Up and down the ladder: The latest comings and goings

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On the cusp of payoffs for patients, stem cell therapy faces threat from unregulated clinics - STAT

Cardiac stem cells from heart disease patients may be harmful – Medical Xpress

June 15, 2017

Patients with severe and end-stage heart failure have few treatment options available to them apart from transplants and "miraculous" stem cell therapy. But a new Tel Aviv University study finds that stem cell therapy may, in fact, harm heart disease patients.

The research, led by Prof. Jonathan Leor of TAU's Sackler Faculty of Medicine and Sheba Medical Center and conducted by TAU's Dr. Nili Naftali-Shani, explores the current practice of using cells from the host patient to repair tissueand contends that this can prove deleterious or toxic for patients. The study was recently published in the journal Circulation.

"We found that, contrary to popular belief, tissue stem cells derived from sick hearts do not contribute to heart healing after injury," said Prof. Leor. "Furthermore, we found that these cells are affected by the inflammatory environment and develop inflammatory properties. The affected stem cells may even exacerbate damage to the already diseased heart muscle."

Tissue or adult stem cells"blank" cells that can act as a repair kit for the body by replacing damaged tissueencourage the regeneration of blood vessel cells and new heart muscle tissue. Faced with a worse survival rate than many cancers, many heart failure patients have turned to stem cell therapy as a last resort.

"But our findings suggest that stem cells, like any drug, can have adverse effects," said Prof. Leor. "We concluded that stem cells used in cardiac therapy should be drawn from healthy donors or be better genetically engineered for the patient."

Hope for improved cardiac stem cell therapy

In addition, the researchers also discovered the molecular pathway involved in the negative interaction between stem cells and the immune system as they isolated stem cells in mouse models of heart disease. After exploring the molecular pathway in mice, the researchers focused on cardiac stem cells in patients with heart disease.

The results could help improve the use of autologous stem cellsthose drawn from the patients themselvesin cardiac therapy, Prof. Leor said.

"We showed that the deletion of the gene responsible for this pathway can restore the original therapeutic function of the cells," said Prof. Leor. "Our findings determine the potential negative effects of inflammation on stem cell function as they're currently used. The use of autologous stem cells from patients with heart disease should be modified. Only stem cells from healthy donors or genetically engineered cells should be used in treating cardiac conditions."

The researchers are currently testing a gene editing technique (CRISPER) to inhibit the gene responsible for the negative inflammatory properties of the cardiac stem cells of heart disease patients. "We hope our engineered stem cells will be resistant to the negative effects of the immune system," said Prof. Leor.

Explore further: Adult stem cell types' heart repair potential probed

More information: Nili Naftali-Shani et al, Left Ventricular Dysfunction Switches Mesenchymal Stromal Cells Toward an Inflammatory Phenotype and Impairs Their Reparative Properties Via Toll-Like Receptor-4Clinical Perspective, Circulation (2017). DOI: 10.1161/CIRCULATIONAHA.116.023527

Journal reference: Circulation

Provided by: Tel Aviv University

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(HealthDay)A new method for delivering stem cells to damaged heart muscle has shown early promise in treating severe heart failure, researchers report online July 27 in Stem Cells Translational Medicine.

Researchers at Karolinska Institutet in Sweden have obtained the first 3D snapshots of a sperm protein attached to a complementary egg coat protein at the beginning of fertilisation. The study, which reveals a common egg ...

As we bask in the summer heat, it is easy to take for granted that humans are also prepared for the cold of winter, with overcoats in the closet and home heating systems ready to be fired up as an added assurance against ...

Organs-on-Chips (Organ Chips) are emerging as powerful tools that allow researchers to study the physiology of human organs and tissues in ways not possible before. By mimicking normal blood flow, the mechanical microenvironment, ...

Monash University's Biomedicine Discovery Institute (BDI) researchers have collaboratively developed a therapeutic approach that dramatically promotes the growth of muscle mass, which could potentially prevent muscle wasting ...

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Researchers from Imperial College London and colleagues have found a potential way to target the receptors that specifically control appetite in mouse brains, potentially without causing other side effects.

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Cardiac stem cells from heart disease patients may be harmful - Medical Xpress

Newly identified method of gene regulation challenges accepted science – Stanford Medical Center Report

The work builds upon a previous study from Barnas laboratory that was published June 1 in Cell. The lead author of that study was postdoctoral scholar Deniz Simsek, PhD. It showed that ribosomes also differ in the types of proteins they accumulate on their outer shells. It also identified more than 400 ribosome-associated proteins, called RAPs, and showed that they can affect ribosomal function.

Every biology student learns the basics of how the genetic code is used to govern cellular life. In broad strokes, the DNA in the nucleus carries the building instructions for about 20,000 genes. Genes are chosen for expression by proteins that land on the DNA and transcribe the DNA sequence into short pieces of mobile, or messenger, RNA that can leave the nucleus. Once in the cells cytoplasm, the RNA binds to ribosomes to be translated into strings of amino acids known as proteins.

Every living cell has up to 10 million ribosomes floating in its cellular soup. These tiny engines are themselves complex structures that contain up to 80 individual core proteins and four RNA molecules. Each ribosome has two main subunits: one that binds to and reads the RNA molecule to be translated, and another that assembles the protein based on the RNA blueprint. As shown for the first time in the Cell study, ribosomes also collect associated proteins called RAPs that decorate their outer shell like Christmas tree ornaments.

Until recently, ribosomes have been thought to take an important but backstage role in the cell, just taking in and blindly translating the genetic code, said Barna. But in the past couple of years there have been some intriguing hints of a more complex scenario. Some human genetic diseases caused by mutations in ribosomal proteins affect only specific organs or tissues, for example. This has been very perplexing. We wanted to revisit the textbook notion that all ribosomes are the same.

In 2011, members of Barnas lab showed that one core ribosomal protein called RPL38/eL38 is necessary for the appropriate patterning of the mammalian body plan during development; mice with a mutation in this protein developed skeletal defects such as extra ribs, facial clefts and abnormally short, malformed tails.

Shi and Fujii used a quantitative proteomics technology called selected reaction monitoring to precisely calculate the quantities, or stoichiometry, of each of several ribosomal proteins isolated from ribosomes within mouse embryonic stem cells. Their calculations showed that not all the ribosomal proteins were always present in the same amount. In other words, the ribosomes differed from one another in their compositions.

We realized for the first time that, in terms of the exact stoichiometry of these proteins, there are significant differences among individual ribosomes, said Barna. But what does this mean when it comes to thinking about fundamental aspects of a cell, how it functions?

To find out, the researchers tagged the different ribosomal proteins and used them to isolate RNA molecules in the act of being translated by the ribosome. The results were unlike what they could have ever imagined.

We found that, if you compare two populations of ribosomes, they exhibit a preference for translating certain types of genes, said Shi. One prefers to translate genes associated with cell metabolism; another is more likely to be translating genes that make proteins necessary for embryonic development. We found entire biological pathways represented by the translational preferences of specific ribosomes. Its like the ribosomes have some kind of ingrained knowledge as to what genes they prefer to translate into proteins.

The findings dovetail with those of the Cell paper. That paper showed that there is more to ribosomes than the 80 core proteins, said Simsek. We identified hundreds of RAPs as components of the cell cycle, energy metabolism, and cell signaling. We believe these RAPs may allow the ribosomes to participate more dynamically in these intricate cellular functions.

Barna and her team have taken a big step toward understanding how ribosomes control protein synthesis by looking at unperturbed stem cells form mammals, said Jamie Cate, PhD, professor of molecular and cell biology and of chemistry at the University of California-Berkeley. They found built-in regulators of translation for a subset of important mRNAs and are sure to find more in other cells. Cate was not involved in the research.

The fact that ribosomes can differ among their core protein components as well as among their associated proteins, the RAPs, and that these differences can significantly affect ribosomal function, highlights a way that a cell could transform its protein landscape by simply modifying ribosomes so that they prefer to translate one type of gene say, those involved in metabolism over others. This possibility would free the cell from having to micromanage the expression levels of hundreds or thousands of genes involved in individual pathways. In this scenario, many more messenger RNAs could be available than get translated into proteins, simply based on what the majority of ribosomes prefer, and this preference could be tuned by a change in expression of just a few ribosomal proteins.

Barna and her colleagues are now planning to test whether the prevalence of certain types of ribosomes shift during major cellular changes, such as when a cell enters the cell cycle after resting, or when a stem cell begins to differentiate into a more specialized type of cell. Theyd also like to learn more about how the ribosomes are able to discriminate between classes of genes.

Although the findings of the two papers introduce a new concept of genetic regulation within the cell, they make a kind of sense, the researchers said.

About 60 percent of a cells energy is spent making and maintaining ribosomes, said Barna. The idea that they play no role in the regulation of genetic expression is, in retrospect, a bit silly.

Other Stanford co-authors are graduate students Kyle Kovary and Naomi Genuth; postdoctoral scholar Hannes Rost, PhD; and Mary Teruel, PhD, assistant professor of chemical and systems biology.

The research was supported by the New York Stem Cell Foundation, the Alfred P. Sloan Foundation, the Mallinckrodt Foundation, a Pew Scholars Award and the National Institutes of Health (a Directors New Innovator Award and grants R21HD086730, R01DK101743, R01DK106241 and P50GM107615).

Stanfords departments of Developmental Biology and of Genetics also supported the work.

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Newly identified method of gene regulation challenges accepted science - Stanford Medical Center Report

Induced Cell Turnover: A proposed modality for in situ tissue regeneration & repair – Medical Xpress

June 15, 2017 Credit: Biogerontology Research Foundation, Feinberg School of Medicine & Swammerdam Institute for Life Sciences

Thursday, July 15, 2017, London, UK: Scientists at the Biogerontology Research Foundation, Feinberg School of Medicine at Northwestern University and Swammerdam Institute of Life Sciences at the University of Amsterdam have published a paper on a proposed method of in situ tissue regeneration called Induced Cell Turnover (ICT) in the journal Human Gene Therapy. The proposed therapeutic modality would aim to coordinate the targeted ablation of endogenous cells with the administration of minimally-differentiated, hPSC-derived cells in a gradual and multi-phasic manner so as to extrinsically mediate the turnover and replacement of whole tissues and organs with stem-cell derived cells.

"One of the major hurdles limiting traditional cell therapies is low levels of engraftment and retention, which is caused in part by cells only being able to engraft at locations of existing cell loss, and by the fact that many of those vacancies have already become occupied by ECM and fibroblasts (i.e. scar tissue) by the time the cells are administered, long after the actual occurrence of cell loss. The crux underlying ICT is to coordinate endogenous cell ablation (i.e. induced apoptosis) with replacement cell administration so as to manually vacate niches for new cells to engraft, coordinating these two events in space and time so as to minimize the ability for sites of cell loss to become occupied by ECM and fibroblasts. This would be done in a gradual and multi-phasic manner so as to avoid acute tissue failure resulting from the transient absence of too many cells at any one time. While the notion of endogenous cell clearance prior to replacement cell administration has become routine for bone marrow transplants, it isn't really on the horizon of researchers and clinicians working with solid tissues, and this is something we'd like to change." said Franco Cortese, Deputy Director and Trustee of the Biogerontology Research Foundation, and lead author on the paper.

Cell-type and tissue-specific rates of induced turnover could be achieved using cell-type specific pro-apoptotic small molecule cocktails, peptide mimetics, and/or tissue-tropic AAV-delivered suicide genes driven by cell-type specific promoters. Because these sites of ablation would still be "fresh" when replacement cells are administered, the presumption is that the patterns of ablation will make administered cells more likely to engraft where they should, in freshly vacated niches where the signals promoting cell migration and engraftment are still active. By varying the dose of cell-type targeted ablative agents, cell type and tissue-specific rates of induced turnover could be achieved, allowing for the rate and spatial distribution of turnover to be tuned to the size of the tissue in order to avoid ablating too many cells at once and inadvertently inducing acute tissue failure.

"Cell therapies are limited by low levels of engraftment, and in principal their ability to improve clinical outcomes is limited by the fact that they can only engraft at locations of existing cell loss. Conversely, therapeutic tissue and organ engineering requires surgery, is more likely to introduce biochemical and mechanical abnormalities to tissue ultrastructure through the decellularization process, and is fundamentally incapable of replacing distributed tissues and structures with a high degree of interconnectivity to other tissues in the body. The aim of ICT is to form a bridge between these two main pillars to regenerative medicine, extending the efficacy of cell therapies beyond a patch for existing cell loss and accomplishing the aim of tissue and organ engineering (i.e. the replacement and regeneration of whole tissues and organs) while potentially remaining free of some of their present limitations." said Giovanni Santostasi, co-author on the paper and a researcher at the Feinberg School of Medicine, Northwestern University.

While future iterations of the therapy could use patient-derived cells, such as ESCs derived via somatic cell nuclear transfer (SCNT) or iPSCs derived from nuclear reprogramming, shorter-term applications would likely use existing stem cell lines immunologically matched to the patient via HLA matching. The authors contend that the cloning of adult organisms with normal lifespans from adult somatic cells testifies to the fact that adult cells can be rejuvenated and used to produce a sufficient quantity of daughter cells to replace the sum of cells constituting adult organisms, and that serial cloning experiments (in which this process is done iteratively, using an adult cell of each subsequent generation to derive the next) attests to this fact even more strongly.

"ICT could theoretically enable the controlled turnover and rejuvenation of aged tissues. The technique is particularly applicable to tissues that are not amenable to growth ex vivo and implantation (as with solid organs)such as the vascular, lymphatic, and nervous systems. The method relies upon targeted ablation of old, damaged and/or senescent cells, coupled with a titrated replacement with patient-derived semi-differentiated stem and progenitor cells. By gradually replacing the old cells with new cells, entire tissues can be replaced in situ. The body naturally turns over tissues, but not all tissues and perhaps not optimally. I am reminded of the quote attributed to Heraclitus: 'No man ever steps in the same river twice, for it's not the same river and he's not the same man.'" said Sebastian Aguiar, a coauthor on the paper and researcher at the Swammerdam Institute of Life Sciences, University of Amsterdam.

"Reversing aging in humans will require a multi-step approach at multiple levels of the organismal organization. In situ targeted ablation of the senescent cells and regeneration will be an important component of comprehensive anti-aging therapies." said Alex Zhavoronkov, Chief Science Officer of the Biogerontology Research Foundation.

The researchers originally proposed ICT in 2016 in the context of biomedical gerontology as a possible means of preventing and/or negating age-related phenotypic deviation for the purposes of healthspan extension, and in this new paper they refine the methodological underpinnings of the approach, take a closer look at potential complications and strategies for their deterrence, and analyze ICT in the context of regenerative medicine as an intervention for a broader range of conditions based on disease or dysfunction at the cellular and intercellular level, with potential utilities absent from traditional cell therapies and tissue/organ engineering, the two main pillars of regenerative medicine. The intervention is still very much conceptual, and any potential utilities over other therapeutic modalities within regenerative medicine would need to be verified via preclinical studies, but their hope is to stimulate further research at this interface between geroscience and regenerative medicine.

More information: Francesco Albert Bosco Cortese et al, Induced Cell Turnover: A novel therapeutic modality for in situ tissue regeneration, Human Gene Therapy (2017). DOI: 10.1089/hum.2016.167

Journal reference: Human Gene Therapy

Provided by: Biogerontology Research Foundation

Researchers at Karolinska Institutet in Sweden have obtained the first 3D snapshots of a sperm protein attached to a complementary egg coat protein at the beginning of fertilisation. The study, which reveals a common egg ...

As we bask in the summer heat, it is easy to take for granted that humans are also prepared for the cold of winter, with overcoats in the closet and home heating systems ready to be fired up as an added assurance against ...

Organs-on-Chips (Organ Chips) are emerging as powerful tools that allow researchers to study the physiology of human organs and tissues in ways not possible before. By mimicking normal blood flow, the mechanical microenvironment, ...

Monash University's Biomedicine Discovery Institute (BDI) researchers have collaboratively developed a therapeutic approach that dramatically promotes the growth of muscle mass, which could potentially prevent muscle wasting ...

As a molecular biologist, Kaori Noridomi gets an up-close view of the targets of her investigations. But when she began studying the molecular structures of a rarely diagnosed autoimmune disorder, myasthenia gravis, she decided ...

Researchers from Imperial College London and colleagues have found a potential way to target the receptors that specifically control appetite in mouse brains, potentially without causing other side effects.

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

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Induced Cell Turnover: A proposed modality for in situ tissue regeneration & repair - Medical Xpress

Pierre Coulombe, Ph.D. to lead UM Department of Cell & Developmental Biology – University of Michigan Health System News (press release)

ANN ARBOR, MI One of the oldest departments at the University of Michigan is about to get a new leader. The U-M Board of Regents today approved the appointment of Pierre A. Coulombe, Ph.D., to lead the Department of Cell and Developmental Biology in the Medical School.

Coulombe will become chair on August 1, and lead one of the nine basic science departments of Michigan Medicine, U-Ms academic medical center. The departments researchers study how structure governs function in cells and tissues throughout the body, and how complex arrays of signals are integrated to foster the proper development of tissues and organs. They also study stem cells, including embryonic stem cells, and train undergraduate, graduate and medical students in cell biology.

The department traces its roots back to 1854, soon after the founding of the Medical School, when it was known as the Department of Anatomy.

Coulombe comes to Michigan from Johns Hopkins University, where he chaired the Department of Biochemistry and Molecular Biology in the Bloomberg School of Public Health for nine years, and held the E.V. McCollum professorship as well as several joint appointments in the School of Medicine. At Hopkins, Coulombe was noted for at recruiting and nurturing junior faculty members to success, and developing robust training programs for graduate students and post-doctoral fellows. He was also instrumental in addressing the departments infrastructure needs.

To me, cell and developmental biology are critically important endeavors as one seeks to translate the wealth of knowledge acquired in biochemistry and molecular biology, along with the power of imaging techniques, into a better understanding of how organs and tissues form, and operate, under normal and disease conditions, he says. This knowledge is also important for developing novel therapies for human disease. U-M already is a formidable institution, and otherwise is making a substantial investment into biomedical research. Therefore, I am absolutely thrilled about the opportunity to lead Cell & Developmental Biology, and team up with my new colleagues in the department and at U-M, to fulfill this potential.

In addition to his appointment in Cell & Developmental Biology, Coulombe will have a joint appointment in the U-M Department of Dermatology. His research focuses on understanding how keratin proteins and the nanoscale filaments they form foster an optimal architecture and function in skin and related epithelia, and how disruption of these processes result in diseases ranging from inherited conditions to cancer.

A native of Montral, Qubec, Coulombe earned his undergraduate degree from the Universit du Qubec Montral and his Ph.D. in Pharmacology from Universit de Montral. He completed his postdoctoral fellowship in the Department of Molecular Genetics and Cell Biology & Howard Hughes Medical Institute at the University of Chicago before joining Johns Hopkins School of Medicine in 1992. He is the author of more than 140 peer-reviewed publications and one book, holds one patent, and has received multiple awards in recognition of his research and teaching endeavors.

For more about the U-M Department of Cell and Developmental Biology, visit https://medicine.umich.edu/dept/cell-developmental-biology.

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Pierre Coulombe, Ph.D. to lead UM Department of Cell & Developmental Biology - University of Michigan Health System News (press release)

Banks targets embryonic stem cell research funding – Fort Wayne Journal Gazette

Rep. Jim Banks, R-3rd, introduced legislation Thursday that would prevent the use of federal funds for stem cell research involving human embryos.

Banks' bill would direct the U.S. Department of Health and Human Services to give priority to medical research with the greatest potential for near-term clinical benefit in human patients and that does not use stem cells from destroyed, discarded or created embryos.

Scientists say embryonic stem cells show potential for transforming into other cells that might repair tissue damaged by disease or injury. Human embryonic stem cells used in research come from donated, unused fertilized eggs developed for in vitro fertilization procedures.

Adult blood stem cells are used to treat leukemia, and adult neural stem cells have been tested for brain disorders and spinal cord injuries.

This bipartisan bill prioritizes stem cell research that has a real impact on patients suffering right now while ensuring that research is conducted ethically without destroying human embryos, Banks, a freshman lawmaker from Columbia City, said in a statement.

Rep. Dan Lipinski, D-Ill., co-sponsored Banks' bill, which is called the Patients First Act of 2017.

The Dickey-Wicker Amendment of 1996 prohibited HHS from funding research using created or destroyed human embryos. But a federal court ruled in 2011 that Dickey-Wicker was ambiguous and did not ban research using stem cells from in vitro fertilization.

The Alliance for Regenerative Medicine, a coalition of medical companies, research institutions and patient advocacy groups that support embryonic stem cell research, had little to say Thursday about Banks' legislation.

As an organization representing the broader global regenerative medicine sector, our position is that we are in favor of government funds supporting the best science in an effort to speed safe and efficacious products to patients in need, Lyndsey Scull, senior communications director for ARM, said in an email.

Scull said ARM would monitor Banks' bill in the legislative process.

Banks' proposal states it would promote the derivation of pluripotent stem cell lines without the creation of human embryos for research purposes and without the destruction or discarding of, or risk of injury to, a human embryo.

The National Institutes of Health defines pluripotent stem cells as those that can give rise to any type of cell in the body except those needed to support and develop a fetus in the womb. They come from embryos and fetal tissue, although induced pluripotent stem cells are genetically reprogrammed cells taken from adult tissues.

In May, Banks led a letter signed by 40 other Republican House members that asked President Donald Trump to replace Dr. Francis Collins as the director of the NIH because of Collins' support for human embryonic stem cell research. Trump announced last week that he is retaining Collins, a geneticist nominated for NIH chief by President Barack Obama and confirmed by unanimous consent by the Senate in 2009.

The NIH is an HHS agency.

bfancisco@jg.net

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Banks targets embryonic stem cell research funding - Fort Wayne Journal Gazette

Pro-Life Congressional Bill Urges NIH to Stop Killing Human Beings in Embryonic Stem Cell Research – LifeNews.com

Republican Congressman Jim Banks (IN-03) today joined with Democratic Congressman Dan Lipinski (IL-03) to introduce a bipartisan bill to direct the Department of Health and Human Services (HHS) and the National Institutes of Health (NIH) to prioritize life-affirming stem cell research with near-term benefits for human patients and to refrain from creating or destroying human embryos in the process.

This bipartisan bill prioritizes stem cell research that has a real impact on patients suffering right now while ensuring that research is conducted ethically with destroying human embryos, said Banks. HHS and NIH both perform important life-saving research and promoting research that protects life has support on both sides of the aisle.

If enacted, the Banks legislation would direct HHS to prioritize stem cell research that has the greatest potential for near-term benefit in human patients. The bill also prohibits such research from creating or destroying human embryonic stem cells in the process.

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The bill would direct the HHS Secretary, in consultation with the Director of the NIH, to publish final guidelines to ensure all future research prioritizes the potential for near-term clinical benefit in human patients while refraining from creating or destroying human embryos in the process. Additionally, the bill would require the HHS Secretary to submit a report each fiscal year outlining the number of stem cell research proposals that were peer reviewed, a summary of all related proposals that were not funded and a subsequent explanation for why they failed to receive funding.

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Pro-Life Congressional Bill Urges NIH to Stop Killing Human Beings in Embryonic Stem Cell Research - LifeNews.com