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Endonovo Therapeutics to Develop Next Generation, Off-The-Shelf, Cell Free Regenerative Products

By Stem Cell Medical Center

SOURCE: Endonovo Therapeutics, Inc.

Dr. Leonard Makowka, a Leading Authority in Hepatic Science, Joins Endonovo Therapeutics as Its Chief Medical Officer

LOS ANGELES, CA--(Marketwired - Jun 11, 2014) - Endonovo Therapeutics, Inc. (OTCQB: ENDV) ("Endonovo" or the "Company"), a biotechnology company developing innovative regenerative therapies, announced today that it has created technologies to pursue the development and manufacturing of next generation regenerative products that no longer require the administration of stem cells. Endonovo's "Cell Free" products will harness the biological molecules secreted by cells to create truly "off-the-shelf" therapeutics that can be delivered to patients in a much timelier manner than is possible with autologous and allogeneic stem cell therapies.

There are currently more than 300 mesenchymal stem cell clinical trials underway to treat a wide range of pathological conditions listed on the ClinicalTrials.gov database. These clinical trials are still based on the transplantation of mesenchymal stem cells. Endonovo is developing therapies that may mitigate many of the current drawbacks of cell-based therapies, such as the low survival and engraftment rate of transplanted stem cells, the risk of occlusion in microvasculature, and unregulated growth, such as hyper-innervation or ossification and/or calcification in the body.

"Cell Free" regenerative products may also mitigate many of the limitations to the commercialization of stem cell therapies, such as the manufacturing, storage and delivery of viable cells. "Cell Free" regenerative products are commercially more attractive because they are simpler to manufacture, easier to store and maintain their therapeutic potency for longer periods of time when compared to stem cells.

Additionally, Endonovo Therapeutics announced that it has appointed Dr. Leonard Makowka, M.D., Ph.D., to serve as the Company's Chief Medical Officer. Dr. Makowka was formerly the Chairman of the Department of Surgery and Director of Transplantation Services at Cedars Sinai Medical Center in Los Angeles, CA, and served as Professor of Surgery at the UCLA School of Medicine. He was also Executive Director of the Comprehensive Liver Disease Center, which used a multiple disciplinary approach to the treatment of liver disease, at St. Vincent's Medical Center in Los Angeles, CA. He has published over 400 articles and chapters in both clinical and basic scientific research and has been regarded as a leading authority in hepatic science. Dr. Makowka has also served as a board member of various public and private healthcare and life science companies.

"We are very pleased to welcome Dr. Makowka to the Endonovo team," said Endonovo Therapeutics CEO, Alan Collier. "His experience and expertise will be a tremendous asset in the development of 'cell-free' therapeutics that can promote the regeneration of tissues without the need to inject stem cells into the body, which would result in safer, more effective therapies, lower costs and the saving of lives."

"I am excited to join Endonovo Therapeutics and help the company develop its 'cell-free' therapeutic platform," stated Dr. Leonard Makowka, Chief Medical Officer at Endonovo Therapeutics. "Although it is early in the developmental lifecycle, we are excited about the development of first-in-class, 'cell free' regenerative products that can protect and stimulate the regeneration of tissues. These products would be truly 'off-the-shelf' therapies that could be quickly administered following organ injuries, such as a heart attack, a stroke, acute and/or chronic liver disease and the 'aging' of organs, in order to protect the organ and begin healing it."

About Endonovo Therapeutics

Endonovo Therapeutics, Inc. is a publicly traded biotechnology company developing off-the-shelf, cell free regenerative products and non-invasive, bioelectronic therapies designed to extend and enhance the human life by regenerating tissues and organs that have become injured or damaged due to disease and age. The Company is developing therapies for various inflammatory, autoimmune and degenerative diseases using biomolecules secreted from cells and Time-Varying Electromagnetic Fields (TVEMF).

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Endonovo Therapeutics to Develop Next Generation, Off-The-Shelf, Cell Free Regenerative Products

Umbilical cord transplants saving lives

By Stem Cell Medical Center

LOS ANGELES (KABC) --

Amanda Canale doesn't take time with her daughter and niece for granted. She's just happy to feel good.

"I've been in the hospital, and I've been sick my whole life," Amanda said.

Amanda was born with a rare blood disorder that required daily shots.

"Basically, I have no white blood cells," Amanda said. "I have no immune system at all."

At 23, she developed leukemia and was given two weeks to live. She desperately needed a bone marrow transplant, but family members weren't matches. Her doctor suggested an umbilical cord blood transplant.

"The cord was a perfect match and it was available, so it was the right solution for her," Edward Agura, MD, Medical Director of Bone Marrow Transplantation, Baylor University Medical Center, Dallas, said.

Cord blood contains stem cells that regenerate. Mothers of newborns can save their child's own blood or donate it. More than 30,000 transplants have been performed worldwide. However, because the blood comes from a tiny newborn, there's not much of it.

"The cord blood is rare, precious and few, and yet is more potent in its ability to grow," Dr. Agura said.

Now, doctors at Baylor are treating patients by combining cord blood from multiple donors. They've found this increases the number of stem cells and provides faster recovery. Amanda's transfusion was from a baby whose mother donated six years earlier. The procedure completely cured her cancer and blood disorder.

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Umbilical cord transplants saving lives

Saved by his cat, heart attack survivor is first Toronto patient in a groundbreaking cell therapy trial

By Stem Cell Clinic

Nurses called him a celebrity, but Robert Clark said, Nah. He wasnt interested in fame or anticipating fortune, but he gladly took the free cab ride home from the hospital.

After a severe heart attack five weeks ago, the 67-year-old has become the first Toronto patient to enter a groundbreaking cell therapy trial led by researchers at St. Michaels Hospital and the Ottawa Hospital Research Institute.

In a world first, the Canadian study uses genetically enhanced stem cells to repair damaged muscle in heart attack survivors. The souped-up cells are the patients own, made young and healthy again fountain-of-youth style in a laboratory. Cell therapy researchers across the globe are monitoring the study, cautiously optimistic that it could lead to a breakthrough the field has long been waiting for.

We all have, circulating in our blood, stem cells that have the potential to repair and regenerate damaged tissues. In people with risk factors for heart disease or in patients who have suffered a heart attack, these cells dont have the same healing capacity, says Dr. Michael Kutryk, a cardiologist at St. Michaels and principal investigator on the study.

The strategy we are using is to restore the bodys own mechanisms to help fix the heart.

The trials first Toronto patient was having a little rest on his couch on April 28 when he started to feel funny.

OK, so I was petting my cat, Clark recalls, and all of a sudden she went meow, meow, meow, and jumped right on my chest, right?

As he tells the story in his 5th-floor Upper Beach apartment, Clark gets down on his hands and knees and yanks Geisha the cat out from under a small cot. Shes a beautiful tabby with saucer eyes as bright and clear a shade of green as her owners are blue.

Clark is not saying the cat gave him a heart attack. Quite the opposite, actually.

She was warning me that something was wrong, he says solemnly, cradling Geisha in his skinny, tattoo-covered arms. (These are older than you are, he says of the faded green ink blots, and its true.)

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Saved by his cat, heart attack survivor is first Toronto patient in a groundbreaking cell therapy trial

Scientists find trigger to decode the genome

By Uncategorized

Scientists from The University of Manchester have identified an important trigger that dictates how cells change their identity and gain specialized functions.

And the research, published in Cell Reports, has brought them a step closer to being able to decode the genome.

The scientists have found out how embryonic stem cell fate is controlled which will lead to future research into how cells can be artificially manipulated.

Lead author Andrew Sharrocks, Professor in Molecular Biology at The University of Manchester, said: "Understanding how to manipulate cells is crucial in the field of regenerative medicine which aims to repair or replace damaged or diseased human cells or tissues to restore normal function."

During the research the team focused on the part of the cellular genome that gives a gene its expression known as the 'enhancer'. This controls the conversion of DNA from genes into useful information that provides the building blocks that determine the structure and function of our cells.

Different enhancers are active in different cell types, allowing the production of distinct gene products and hence a range of alternative cell types. In the current study, the team have determined how these enhancers become active.

Professor Sharrocks said: "All of us develop into complex human beings containing millions of cells from a single cell created by fertilization of an egg. To transit from this single cell state, cells must divide and eventually change their identity and gain specialised functions. For example we need specific types of cells to populate our brains, and our recent work has uncovered the early steps in the creation of these types of cells.

"One of the most exciting areas of regenerative medicine is the newly acquired ability to be able to manipulate cell fate and derive new cells to replace those which might be damaged or lost, either through old age or injury. To do this, we need to use molecular techniques to manipulate stem cells which have the potential to turn into any cell in our bodies."

But one of the current drawbacks in the field of regenerative medicine is that the approaches are relatively inefficient, partly because scientists do not fully understand the basic principles which control cell fate determination.

"We believe that our research will help to make regenerative medicine more effective and reliable because we'll be able to gain control and manipulate cells -- thus our understanding of the regulatory events within a cell shed light on how to decode the genome," concluded Professor Sharrocks.

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Scientists find trigger to decode the genome

Jordans stem-cell law can guide the Middle East

By Uncategorized

In January, Jordan passed a law to control research and therapy using human stem cells derived from embryos the first such regulation in the Arab and Islamic region. I was part of the group headed by Abdalla Awidi Abbadi, director of the Cell Therapy Center at the University of Jordan in Amman, that initiated the call for the law and later drafted it. Stem-cell research is a hot topic for Jordan because of the kingdoms status as a health-care hub that draws patients from abroad. It is already one of few countries in the Middle East with regulations for protecting people who participate in clinical trials. This latest law should serve as an example to other countries in the region.

The new rules ban private companies from using human embryonic stem (ES) cells in research or therapies. Such work will be allowed only in government organizations or publicly funded academic institutions in Jordan, which have higher levels of transparency than private firms and are supervised by the health ministry and a specialized committee. The law also bans payment for donations of stem cells and eggs, and says that modified and manipulated cells are not to be used for human reproduction. There is no current research on human ES cells in Jordan; this is a pre-emptive step.

Much of the controversy and disagreement over work on stem cells worldwide arises from the different views of the major religions on the earliest stages of life. Although the use of human ES cells is opposed by the Roman Catholic Church and some Protestant denominations, it is generally supported by the Jewish community and accepted in many Muslim countries. There is no consensus on when human embryonic life begins, but the majority of Muslim scholars consider it to start 40120 days after conception and therefore hold the view that a fertilized egg up to 5days old has no soul it is not human life but biological life. So for many, there is no ethical problem in the Islamic faith with using an early embryo to produce stem cells.

All our discussions in Jordan have concluded that stem-cell research is permissible in Islam.

Such conclusions are not easy to reach. Many Muslim countries consider legislation and bioethics principles to be based on three pillars of Islamic law. The first is the Quran. The second is Sunnah, or the legislative decisions of the Prophet Muhammad. The third is ijmaa the consensus of Muslim scholars and ijtihad, the concept that every adequately qualified scholar has the right to independently solve problems. On the basis of these pillars, Iran, Saudi Arabia and Tunisia have drawn up guidelines on stem-cell research, but they are not legally binding.

Jordans stem-cell law is the product of years of discussions by committees comprising scientists, physicians, Arabic-language experts, lawyers and Muslim and Christian theologians. The issues that arose confusion between stem cells and embryonic stem cells, for instance were discussed and resolved. We consulted with both the National Committee for Science and Technology Ethics and the education ministry. The final law was approved by the council of Muslim scholars, the Majlis Al-Iftaa.

The council agreed with a 2003 decision (fatwa) by Muslim scholars that allows the use of human ES cells from permissible sources including legally produced excess fertilized eggs from invitro fertilization. The decision to ban private companies from using these cells was driven by concerns that the work would encourage termination of pregnancies, which is illegal in Jordan unless the mothers life or health is at risk. The council was clear that the new law must forbid human reproductive cloning and should not allow embryos to be created from the sperm and eggs of unmarried couples.

The distinction drawn between the various sources of stem cells earlier in the discussion process allowed the Majlis Al-Iftaa to take a more permissive approach to techniques using stem cells that are not derived from human embryos. For example, somatic-cell nuclear transfer (in which a patients DNA is transplanted into an unfertilized human egg that has no nucleus) and induced pluripotent stem cells, which are made from adult cells, can be worked on by the private sector under the new rules.

The therapeutic use of bone-marrow transplantation including transplants of blood-forming stem cells is well established in Jordan. Such procedures are already regulated by existing laws on medical practice, so the new law makes a clear distinction between these techniques and human ES-cell therapy.

The legislation not only covers all current aspects of stem-cell research and use, but also leaves room for later modification. It mandates the creation of a national committee that, among other things, will take responsibility for laying out specific regulations for stem-cell banking in accordance with international standards.

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Jordans stem-cell law can guide the Middle East

Fetal-cell revival for Parkinsons

By Stem Cell Treatment

ANIMATED HEALTHCARE LTD/SPL

Defective brain neurons are responsible for the mobility problems seen in people with Parkinsons disease.

A neurosurgery team will next month transplant cells from aborted human fetuses into the brain of a person with Parkinsons disease. The operation breaks a decade-long international moratorium on the controversial therapy that was imposed after many patients failed to benefit and no one could work out why.

But the trial comes just as other sources of replacement cells derived from human stem cells are rapidly approaching the clinic. And this time, scientists want to make sure that things go better. So the teams involved in all the planned trials have formed a working group to standardize their research and clinical protocols in the hope that their results will be more easily interpretable.

People with Parkinsons disease suffer from a degeneration of neurons that produce the neurotransmitter dopamine, which is crucial for normal movement. This often leaves patients with severe mobility problems. Standard treatment includes the drug l-dopa, which replaces dopamine in the brain but can cause side effects. The cellular therapies aim to replace the missing neurons with dopamine-producing (dopaminergic) cells from fetal brains or with those derived from human stem cells.

The moratorium on replacement-therapy trials was introduced in 2003 because the early fetal-cell studies had produced varying results that were impossible to interpret.

We want to avoid a repeat of this situation, says neurologist Roger Barker at the University of Cambridge, UK, who helped to organize the working groups inaugural meeting in London last month. The group, known as the Parkinsons Disease Global Force, includes scientists from the European, US and Japanese teams about to embark on the trials. At the meeting, they pledged to share their knowledge and experiences.

The first human transplantation of fetal brain cells took place in 1987 at Lund University in Sweden, where the technique was pioneered. Surgical teams took immature fetal cells destined to become dopaminergic neurons from the midbrain of aborted fetuses and transplanted them into the striatum of patients brains, the area of greatest dopamine loss in Parkinsons disease.

More than 100 patients worldwide received the therapy as part of clinical trials before the moratorium. But centres used different procedures and protocols it was impossible to work out why some patients did very well and others didnt benefit at all, says Barker.

In 2006, Barker, together with neuroscientist Anders Bjrklund at Lund University, set up a network to bring together the original seven teams that had performed the transplants, to assess all protocol details and patient data retrospectively.

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Fetal-cell revival for Parkinsons

New Stem Cell Based Treatment for COPD; Nebulized Pure PRP System Uses Blood Growth Factors That Can Trigger Healing …

By Stem Cell Treatment

Sarasota, FL (PRWEB) June 12, 2014

Nebulized Pure PRP may offer COPD sufferers a less expensive and an effective alternative to stem cell therapy. When normal injury occurs, platelets are stimulated to release growth factors, cytokines and other immune system components in what is called the inflammatory phase of healing. In the lungs, platelets can adhere to injured or inflamed endothelial cells where they start the healing process. It is believed that by increasing the number of platelets in the lungs through this method, it is possible to decrease inflammation and accelerate the healing process in the lungs. Platelets are vehicles for the delivery of growth factors (PDGF, TGF-, IGF, EGF, VEGF) that induce proliferation of fibroblasts, osteoblasts and endothelial cells, promoting and accelerating healing of hard and soft tissues.

Autologous Platelet Rich Plasma also contains fibrin, fibronectin and vitronectin that act as cell adhesion molecules for lung epithelial migration. Autologous Platelet Rich Plasma treatment has been evaluated in various medical disciplines including orthopaedics, wound healing, neurosurgery, dentistry as well as cosmetic, plastic and cardiothoracic surgery. Nebulized Pure PRP treatment holds much promise and is being researched for its applications.

This new medical advance can bring effective and affordable healthcare to many patients with COPD. It is also attractive because the patients own blood is used thus, limiting the potential for disease transmission.

Our key product differentiation is to enable the Pure PRP treatment to be applied to patients who are suffering from COPD. COPD is the most dangerous disease in the elderly, affecting more than 200 million people across the globe. COPD is considered to be the cause of about 3 million deaths annually. This is a life-threatening disease caused by many reasons such as smoking, pollution, dust, irritants, genetic disorders, etc. It is associated with the excess production of sputum and an inflammation which obstructs the airways and results in breathing problems.

Though there is no cure for COPD, the condition can be controlled with the help of treatments. Stem cell therapy which has proved to be one of the most successful treatments for many chronic health conditions like heart disease, stroke, osteoporosis, etc., has given a ray of hope in favor of COPD. Stem cells are known for their regenerative properties which help in the development of the tissues and blood cells. These cells are of two types: embryonic stem cells and adult stem cells. Embryonic stem cells can be derived from blastocyst which is a type of embryo; whereas adult stem cells are found in the bone marrow, skin, umbilical cord, placenta and many other tissues. Embryonic stem cells are derived and are grown in cell culture for research and development. But adult stem cells, once removed from the body, divide with great difficulty which makes the treatment difficult to perform. The stem cells are either from the person itself who needs it which is known as autologous stem cell or they can be received from a donor which is known as allogeneic stem cell.

Cells donated by the donor may or may not be accepted by the bodys immune system. Hence, using ones own stem cells reduces the chances of rejection. In COPD, the tissues and cells of the lungs are destroyed, which causes various types of complications. Hence, with the help of stem cell therapy, the destroyed or damaged cells can be regenerated and new lung tissues can be formed. According to the procedure followed by the International Stem Cell Institute (ISCI); San Diego, California, adipose tissue is removed from the patient and is processed with a combination of platelet rich plasma which contains growth factors that help in the process of cell multiplication and development. This helps in COPD treatment as whenever the lungs need repair, about 80% of the stem cells reach the repairing site through the circulatory system. When the blood passes through the lungs, stem cells get trapped in the space where there is damage. The stem cells then start multiplying and repairing the tissue. The recovery does not take place immediately, but improvement can be noticed in 3 to 6 months. It helps in the suppression of inflammation, improves breathing and cures many pulmonary complications. Our Nebulized Pure PRP System aims to support this proposition to treat COPD patients. Treatments run about $1,000 and insurance does not currently pay for this treatment.

Contact our office at (941) 330-8553 to find out more about how Nebulized Pure PRP can offer you relief from symptoms of COPD. Also we are at http://advancedwellness.us/blog2/nebulized-platelet-rich-plasma-prp-for-asthma-copd-and-systemic-growth-effects-in-athletics/

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New Stem Cell Based Treatment for COPD; Nebulized Pure PRP System Uses Blood Growth Factors That Can Trigger Healing ...

Texas Biomed Regenerative Medicine Program Expands With Two New Research Scientists

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Newswise San Antonio, June 10, 2014 Texas Biomedical Research Institute has recruited two new research scientists to its Southwest National Primate Research Center (SNPRC) who will focus on regenerative medicine, working with animal models to develop human stem cell therapies for medical conditions such as Parkinsons disease, degenerative diseases of the eye and muscular dystrophy.

Tiziano Barberi, PhD and Marcel M. Daadi, PhD join Texas Biomed as Associate Scientists in the SNPRC. Barberi comes from the Australian Regenerative Medicine Institute at Monash University in Melbourne, Australia and Daadi arrives from Palo Alto, CA where he was part of the Consulting Faculty of Stanford Universitys Department of Neurosurgery. He is also President and Chief Scientific Officer of NeoNeuron LLC.

Dr. Barberi and Dr. Daadi are significant additions to our regenerative medicine research program, Texas Biomed President and CEO Kenneth P. Trevett said. Both have focused on stem cell research, have published significant research results in peer review journals and received recognition for their leading roles within research teams and at institutions. Regenerative medicine is a major focus for Texas Biomed, where we have new facilities and financial resources dedicated for that purpose, he said. We also look to expand our work with other institutions and groups in San Antonio to promote progress in this field. Dr. Barberi and Dr. Daadi both have strong backgrounds in developing collaborative efforts, and we look forward to the contributions they will make in this important research arena.

Barberi, a native of Italy, had been one of 15 Chief Investigators of the Stem Cells Australia Consortium for stem cell research and Group Leader for the Australian Regenerative Medicine Institute. With a laboratory research focus on the directed differentiation of human pluripotent stem cells (hESC and iPSC) into specific developmental fates, his research aims are to provide tools for human development studies, in vitro disease modeling and a cell therapeutics approach to disease. He described in a seminal work a method to obtain all the clinically relevant neuronal subtypes from mESC, and was the first to have directed differentiation of hESC into mesenchymal precursors and into the progenitor cells forming the skeletal muscle system.

Prior to his work in Australia, Barberi was head of the Laboratory of Stem Cells and Development at the Beckman Research Institute of City of Hope in Duarte, CA. During the time spent at City of Hope, Barberi was awarded the prestigious New Faculty Award from the California Institute for Regenerative Medicine (CIRM). He is an invited reviewer for a number of stem cell-related research journals and is a grant reviewer/assessor for research programs in Canada, Australia, New Zealand and the European Union.

Daadi has unique academia and industry experiences bridging basic and translational research. He comes to Texas Biomed from the San Francisco bay area where he founded a biotechnology company, NeoNeuron, focused on developing therapies for treating neurological disorders. He served as Director of Stem Cell Research, CIRM Disease Team Stroke Neural Transplant Program at Stanford University School of Medicine and Director of the Parkinson's Disease Program at the Sanford Burnham Medical Research Institute, Layton Biosciences Inc and NeuroSpheres LLC.

At Stanford University, Daadi developed a novel technology to purify homogenous populations of neural stem cells from human pluripotent stem cells and coax them to specific types of neurons that can be used for brain repair. His research is paving the way for clinical trials to treat patients with devastating neurological disorders, such as Parkinsons disease, stroke and traumatic brain injury. He seeks to expand on the capabilities of the SNPRC and to build new collaborative programs and projects in stem cell research with colleagues at the University of Texas Health Science Center at San Antonio and the University of Texas at San Antonio.

Daadi serves as editor and reviewer for many peer review journals. He is a permanent member on the National Institutes of Health Grant Review Committee, The Maryland Stem Cell Research Fund and serves on many other national and international Grant Review Committees.

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Texas Biomed Regenerative Medicine Program Expands With Two New Research Scientists

Stem cells edited to produce an HIV-resistant immune system

By Stem Cell Doctors

A team of haematologists has engineered a particular white blood cell to be HIV resistant after hacking the genome of induced pluripotent stem cells (iPSCs).

The technique has been published in the Proceedings of the National Academy of Sciences and was devised by Yuet Wai Kan of the University of California, former President of the American Society of Haematology, and his peers.

The white blood cell the team had ideally wanted to engineer was CD+4 T, a cell that is responsible for sending signals to other cells in the immune system, and one that is heavily targeted by the HIV virus. When testing for the progress of HIV in a patient, doctors will take a CD4 cell count in a cubic millimetre of blood, with between 500 and 1,500 cells/mm3 being within the normal range. If it drops below around 250, it means HIV has taken hold -- the virus ravages these cells and uses them as an entry point.

HIV gains entry by attaching itself to a receptor protein on the CD+4 Tcell surface known as CCR5.If this protein could be altered, it could potentially stop HIV entering the immune system, however. A very small number of the population have this alteration naturally and are partially resistant to HIV as a result -- they have two copies of a mutation that prevents HIV from hooking on to CCR5 and thus the T cell.

In the past, researchers attempted to replicate the resistance by simply transplanting stem cells from those with the mutation to an individual suffering from HIV. The rarity of this working has been demonstrated by the fact that just one individual,Timothy Ray Brown(AKA the Berlin patient), has been publicly linked to the treatment and known to be HIV free today. The Californian team hoped to go right to the core of the problem instead, and artificially replicate the protective CCR5mutation.

Kan has been working for years on a precise process for cutting and sewing back together genetic information. His focus throughout much of his career has been sickle cell anaemia, and in recent years this has translated to researching mutations and how these can be removed at the iPSC stage, as they are differentiated into hematopoietic cells. He writes on his university web page: "The future goal to treatment is to take skin cells from patients, differentiate them into iPS cells, correct the mutations by homologous recombination, and differentiate into the hematopoietic cells and re-infuse them into the patients. Since the cells originate from the patients, there would not be immuno-rejection." No biggie.

This concept has now effectively been translated to the study of HIV and the CD+4 T cell.

Kan and his team used a system known as CRISPR-Cas9 to edit the genes of the iPSCs. It uses Cas9, a protein derived from bacteria, to introduce a double strand break somewhere at the genome, where part of the virus is then incorporated into the genome to act as a warning signal to other cells. An MIT team has already used the technique to correct a human disease-related mutation in mice.

When Kan and his team used the technique they ended up creating HIV resistant white blood cells, but they were not CD+4 T-cells. They are now speculating that rather than aiming to generate this particular white blood cell with inbuilt resistance, future research instead look at creating HIV resistant stem cells that will become all types of white blood cells in the body.

Of course, with this kind of therapy the risk is different and unexpected mutations could occur. In an ideal world, doctors will not want to be giving constant cell transplants, but generating an entirely new type of HIV resistant cells throughout the body carries its own risks and will need stringent evaluation if it comes at all close to being proven.

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Stem cells edited to produce an HIV-resistant immune system

Mount Sinai Researchers Identify Protein That Keeps Blood Stem Cells Healthy as They Age

By Stem Cell Medicine

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Newswise (New York June 9, 2014) -- A protein may be the key to maintaining the health of aging blood stem cells, according to work by researchers at the Icahn School of Medicine at Mount Sinai recently published online in Stem Cell Reports. Human adults keep stem cell pools on hand in key tissues, including the blood. These stem cells can become replacement cells for those lost to wear and tear. But as the blood stem cells age, their ability to regenerate blood declines, potentially contributing to anemia and the risk of cancers like acute myeloid leukemia and immune deficiency. Whether this age-related decline in stem cell health is at the root of overall aging is unclear.

The new Mount Sinai study reveals how loss of a protein called Sirtuin1 (SIRT1) affects the ability of blood stem cells to regenerate normally, at least in mouse models of human disease. This study has shown that young blood stem cells that lack SIRT1 behave like old ones. With use of advanced mouse models, she and her team found that blood stem cells without adequate SIRT1 resembled aged and defective stem cells, which are thought to be linked to development of malignancies.

"Our data shows that SIRT1 is a protein that is required to maintain the health of blood stem cells and supports the possibility that reduced function of this protein with age may compromise healthy aging," says Saghi Ghaffari, MD, PhD, Associate Professor of Developmental and Regenerative Biology at Mount Sinai's Black Family Stem Cell Institute, Icahn School of Medicine. "Further studies in the laboratory could improve are understanding between aging stem cells and disease."

Next for the team, which includes Pauline Rimmel, PhD, is to investigate whether or not increasing SIRT1 levels in blood stem cells protects them from unhealthy aging or rejuvenates old blood stem cells. The investigators also plan to look at whether SIRT1 therapy could treat diseases already linked to aging, faulty blood stem cells.

They also believe that SIRT1 might be important to maintaining the health of other types of stem cells in the body, which may be linked to overall aging.

The notion that SIRT1 is a powerful regulator of aging has been highly debated, but its connection to the health of blood stem cells "is now clear," says Dr. Ghaffari. "Identifying regulators of stem cell aging is of major significance for public health because of their potential power to promote healthy aging and provide targets to combat diseases of aging," Dr. Ghaffari says.

Researchers from Harvard Medical School and Children's Hospital in Boston participated in the study.

About the Mount Sinai Health System The Mount Sinai Health System is an integrated health system committed to providing distinguished care, conducting transformative research, and advancing biomedical education. Structured around seven member hospital campuses and a single medical school, the Health System has an extensive ambulatory network and a range of inpatient and outpatient servicesfrom community-based facilities to tertiary and quaternary care.

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Mount Sinai Researchers Identify Protein That Keeps Blood Stem Cells Healthy as They Age

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