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New disease gene discovery sheds light on cause of bone marrow failure

PUBLIC RELEASE DATE:

6-Feb-2014

Contact: Charli Scouller c.scouller@qmul.ac.uk 07-709-825-741 Queen Mary, University of London

The study, published in The American Journal of Human Genetics, detected and identified a new disease gene (ERCC6L2). In its normal form, the gene plays a key role in protecting DNA from damaging agents, but when the gene is mutated the cell is not able to protect itself in the normal way.

The research findings suggest that the gene defect and the subsequent DNA damage was the underlying cause of bone marrow failure among the study participants.

Bone marrow failure is a term used for a group of life threatening disorders associated with an inability of the bone marrow to make an adequate number of mature blood cells.

Patients were recruited from all over the world to join an international bone marrow failure registry and researchers used new DNA sequencing technologies to study cases of bone marrow failure with similar clinical features. These included bone marrow failure associated with neurological abnormalities (learning defects and developmental delay), and patients whose parents were first cousins.

The findings mean it is now possible to carry out a reliable genetic test (including antenatal testing) in these families and get an accurate diagnosis. In the long term, with further research, the findings could lead to the development of new treatment for this specific gene defect.

Professor Inderjeet Dokal, Chair of Paediatrics and Child Health at Queen Mary University of London, comments:

"New DNA sequencing technology has enabled us to identify and define a new gene defect which causes a particular type of bone marrow failure. This is a promising finding which we hope one day could lead to finding an effective treatment for this type of gene defect. Clinicians treating patients with bone marrow failure should now include analysis for this gene in their investigation.

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First FSH Muscular Dystrophy High-Throughput Drug Discovery Study Published

Lexington, MASS (PRWEB) February 06, 2014

Facioscapulohumeral muscular dystrophy (FSHD) is among the most common muscle-wasting diseases, affecting more than 500,000 people around the world. Its cause is genetic, passed from generation to generation, although 30 percent of cases arise spontaneously in families with no previous history. There is no treatment, but in a new study published in the journal, Skeletal Muscle on February 1, 2014, researchers have identified dozens of compounds showing early promise for future treatments.

This is the first published high-throughput drug screening study for FSHD, noted June Kinoshita, Executive Director of the FSH Society, which helped to fund the research. Years of investment in basic research to understand the genetic mechanism of the disease and to develop cell-based assays have made it possible to carry out this efficient strategy to identify drug candidates.

Recent discoveries point to a mysterious protein called DUX4 as a lead suspect in causing FSHD. Normally suppressed in adult muscles, DUX4 is unleashed in FSHD, with toxic effects on muscle cells. In people with FSHD, the facial (facio), shoulder (scapula) and upper arm (humeral) muscles are prone to degenerate, giving the disease its name. FSHD can also affect the lower abdomen and legs, leading to profound disability.

To hunt for drugs that can stop DUX4, the research team, led by Michael Kyba, PhD, of the University of Minnesota, engineered mouse myoblasts (immature muscle cells) to express DUX4 under the control of a genetic switch that is triggered by adding the antibiotic doxycycline to the petri dish.

When DUX4 is switched on, the cells begin to die, and they also become more vulnerable to a variety of chemical insults. Drugs were added to the cultured cells to see if any of them rescued the cells.

The research team tested thousands of compounds on these DUX4-expressing cells, including 1,120 Food and Drug Administration-approved off-patent drugs and 43,000 other chemicals. After extensive studies to weed out false positives, and further winnowing to select compounds with favorable chemical properties, the investigators honed the list to 52 hits, or candidates.

Remarkably, two-thirds of our hits are compounds that protect cells from oxidative stress, Kyba said. Although we need to be cautious extrapolating from cells in a dish to human patients, I am enthusiastic about testing whether protecting cells from oxidative stress is beneficial in FSHD.

Further work is now being planned to understand the precise mechanism behind the anti-DUX4 activity of each of these compounds. This research will help the investigators focus on the most promising ones to develop into therapies. At the same time, each compound can bring to light new insights into how DUX4 causes this devastating disease.

We were very, very fortunate to get support from the FSH Society in the form of a postdoctoral fellowship back in the dark ages when nobody else was funding research on DUX4, said Kyba. Additional funding came from the National Institutes of Health, Dr. Bob and Jean Smith Foundation, Friends of FSH Research, the FSHD Global Research Foundation and the Muscular Dystrophy Association.

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First FSH Muscular Dystrophy High-Throughput Drug Discovery Study Published

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Critical factor (BRG1) identified for maintaining stem cell pluripotency

PUBLIC RELEASE DATE:

6-Feb-2014

Contact: Vicki Cohn vcohn@liebertpub.com 914-740-2100 x2156 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, February 6, 2014The ability to reprogram adult cells so they return to an undifferentiated, pluripotent statemuch like an embryonic stem cellis enabling the development of promising new cell therapies. Accelerating progress in this field will depend on identifying factors that promote pluripotency, such as the Brg1 protein described in a new study published in BioResearch Open Access, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the BioResearch Open Access website.

In "BRG1 Is Required to Maintain Pluripotency of Murine Embryonic Stem Cells," Nishant Singhal and coauthors, Max Planck Institute for Molecular Biomedicine, Mnster, and University of Mnster, Germany, demonstrate the critical role the Brg1 protein plays in regulating genes that are part of a network involved in maintaining the pluripotency of embryonic stem cells. This same network is the target for methods developed to reprogram adult somatic cells.

"This work further clarifies the role of the Brg1 containing BAF complex in regulating pluripotency and has important implications for all cellular reprogramming technologies," says BioResearch Open Access Editor Jane Taylor, PhD, MRC Centre for Regenerative Medicine, University of Edinburgh, Scotland.

###

About the Journal

About the Publisher

Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in promising areas of science and biomedical research, including, DNA and Cell Biology, Tissue Engineering, Stem Cells and Development, Human Gene Therapy, HGT Methods, and HGT Clinical Development, and AIDS Research and Human Retroviruses. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 80 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.

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FluidX Capper/Decapper – Video


FluidX Capper/Decapper

By: Center for Genetic Medicine

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JANUS MDT – Video


JANUS MDT

By: Center for Genetic Medicine

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JANUS MDT - Video

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Public divided on genetic testing to predict cancer risk …

A national poll from the University of Utah's Huntsman Cancer Institute shows 34 percent of respondents would not seek genetic testing to predict their likelihood of developing a hereditary cancer -- even if the cost of the testing was not an issue. Concerns about employment and insurability were cited as the primary reason, even though current laws prohibit such discrimination.

The poll also shows only 35 percent of respondents would be extremely or very likely to seek aggressive prophylactic or preventive treatment, such as a mastectomy, if they had a family history of cancer and genetic testing indicated a genetic pre-disposition to cancer.

"I see patients every week who could have taken steps to reduce their risk if they'd known they'd had a predisposition for a certain type of cancer. The best treatment for cancer is prevention, of which genetic testing plays an integral role," said Saundra Buys, M.D., co-director of the Family Cancer Assessment Clinic and medical director of the High Risk Cancer Research at Huntsman Cancer Institute (HCI), and professor of medicine at the University of Utah. "In addition to educating the public about the important role genetic testing plays in both prevention and treatment of cancer, we must also work to eliminate perceived false barriers to testing, such as concerns about insurability and employment."

Nearly 40 percent of those who said they wouldn't seek testing reported being somewhat or extremely concerned that the results would impact opportunities for employment, while 69 percent of that same group reported being somewhat or extremely concerned that the results would have an adverse impact on their ability to get insurance.

Inherited mutations play a major role in the development of approximately 5 percent of all cancers. Genetic mutations associated with more than 50 hereditary cancer syndromes -- including those discovered at the University of Utah for melanoma, colon and breast cancer -- have been identified.

Buys says the survey demonstrates that even with increased media attention to genetic testing in recent months more work is needed to educate the public about the type of information genetic testing provides and who should seek it. She says family and personal health history are the most important factors in determining whether a person should consider genetic testing.

She warns, however, that genetic testing is only as good as the genetic counseling that accompanies it. "There are many genetic tests being ordered in physician offices around the country without the benefit of genetic counseling. The results of these tests are complex, and without appropriate counseling, can cause confusion and unneeded anxiety for patients," said Buys.

Other findings from the poll:

Following recommended screenings: 63 percent of respondents reported being extremely or very likely to follow all recommended screenings if they knew there was a history of cancer in their family.

Testing to help identify best course of treatment: 85 percent of respondents stated that if diagnosed with cancer they would be willing to undergo genetic testing if it could help determine the most effective course of treatment.

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Genetics Hens , and the problem of eating there Eggs – Video


Genetics Hens , and the problem of eating there Eggs

By: Ray wasp

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Genetics Hens , and the problem of eating there Eggs - Video

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Building Better Genetics – Schiefelbein Farms – Video


Building Better Genetics - Schiefelbein Farms
Cattlemen like Minnesota Angus breeder Don Schiefelbein rely on DNA information from the Zoetis HD 50K test and genomic-enhanced EPDs to make the best possib...

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Introduction MODY and GDM Genetics – Video


Introduction MODY and GDM Genetics
Watch this movie on Plotagon: http://plotagon.com/3810.

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Alliance for Cancer Gene Therapy (ACGT) Targets Brain, Pediatric Cancers with $1 Million in New Grants

Stamford, CT (PRWEB) February 06, 2014

Alliance for Cancer Gene Therapy (ACGT) the nations only non-profit dedicated exclusively to cell and gene therapies for cancer is redoubling its efforts to treat and combat cancers in the New Year, and announces $1 million in recent grants.

The funding spread across three grants will support basic and clinical science at premier institutions in and outside the United States, with ACGTs mission top-of-mind: uncovering effective, innovative cancer treatments that supersede radiation, chemotherapy and surgery.

This January, ACGTs President and Co-Founder Barbara Netter has announced two Young Investigator Grants that provide promising researchers with $250,000 each for two- to three-year studies.

Young Investigator Fan Yang, PhD Assistant Professor of Orthopedic Surgery and Bioengineering at Stanford University will use the funds to research new treatment options for pediatric brain cancer, the leading cause of death from childhood cancer. Dr. Yangs study will deploy adult-derived stem cells to target solid brain tumor cells, which are often highly invasive and difficult to treat.

Arnob Banerjee, MD, PhD Assistant Professor of Hematology and Oncology at the University of Maryland will use ACGTs funding to further develop the long-term effectiveness of immune-mediated treatments, the most advanced form of gene therapy.

It is imperative that the best and brightest young scientists like Fan Yang and Arnob Banerjee have the funds necessary to study and treat cancer, Netter said. This was my husband Edwards vision in 2001, when gene cell therapy was a fledgling science. We are proud to continue his pioneering foresight today. Partnerships with Dr. Yang, a former fellow at MIT, and Dr. Banerjee, a former fellow and instructor at the University of Pennsylvania, dovetail with ACGTs record of funding outstanding researchers and physicians with the capability to make unprecedented breakthroughs.

The Young Investigator grants come on the heels of a $500,000 Investigators Award to John Bell, PhD, Senior Research Scientist and Professor of Medicine at the Ottawa Hospital Research Institute in Canada. Dr. Bell has worked extensively with oncolytic viruses man-made viruses that target only cancer cells, and spare patients the harrowing side-effects of chemotherapy, radiation or surgery and has discovered the enormous promise they offer in the war on cancer.

The research and trials funded by ACGTs grant have the potential to treat metastatic and recurrent brain cancer, extend patients survival timeline, and vastly improve patients quality of life during treatment, Dr. Bell said.

ACGT has served as a major funding engine in the fight against cancer since its formation in 2001, and has provided nearly $25 million in grants to date. ACGT was created by Barbara and Edward Netter after the loss of their daughter-in-law to breast cancer. Since Edwards passing in 2011, Barbara Netter has led the foundation as President and Co-Founder, continuing her husbands vision.

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Alliance for Cancer Gene Therapy (ACGT) Targets Brain, Pediatric Cancers with $1 Million in New Grants

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ASH 2013: The Future of Genomics & Personalized Medicine: Genomics & Blood Cancers – Video


ASH 2013: The Future of Genomics Personalized Medicine: Genomics Blood Cancers
Joining Vital Options CEO, Selma Schimmel -- The Future of Genomics Personalized Medicine : Keith Stewart, MD, Mayo Clinic; Anne Quinn Young, MPH, Multiple...

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ASH 2013: The Future of Genomics & Personalized Medicine: Closing Thoughts From the Panel – Video


ASH 2013: The Future of Genomics Personalized Medicine: Closing Thoughts From the Panel
Joining Vital Options CEO, Selma Schimmel -- The Future of Genomics Personalized Medicine : Keith Stewart, MD, Mayo Clinic; Anne Quinn Young, MPH, Multiple...

By: Vital Options International / The Group Room

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Transfer – Spinal cord injury – Video


Transfer - Spinal cord injury
513212 OT-CMU.

By: Chocco Ba

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Transfer - Spinal cord injury - Video

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Tom Bolewski Walking With the C.O.R.E. Team After Spinal Cord Injury in February 2014 – Video


Tom Bolewski Walking With the C.O.R.E. Team After Spinal Cord Injury in February 2014

By: Center of Restorative Exercise

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Global Stem Cells Group, Inc. and BioHeart, Inc. Launch Clinical Trial for COPD Stem Cell Therapies

Miami (PRWEB) February 05, 2014

Global Stem Cells Group, Inc. and BioHeart, Inc. announce the launch of a clinical trial for the treatment of Chronic Obstructive Pulmonary Disease (COPD) using adipose-derived stem cell technology. The clinical trials will be held at the Global Stem Cells treatment center in Cozumel, Mexico, as well as in several U.S. states. Global Stem Cells Group affiliate Regenestem in collaboration with CMC Hospital of Cozumel offer cutting-edge cellular medicine treatments to patients from around the world

The study titled "An Open-label, Non-Randomized, Multi-Center Study to Assess the Safety and Effects of Autologous Adipose-Derived Stromal Cells Delivered intravenously in Patients with Chronic Obstructive Pulmonary Disease" is lead by principal investigator Armando Pineda Velez, Global Stem Cells Group Medical Director. Global Stem Cells Group has represented that it offers the most advanced protocols and techniques in cellular medicine from around the world.

The Cozumel clinical trials will be lead by Rafael Moguel, M.D., an advocate and pioneer in the use of stem cell therapies to treat a wide variety of conditions.

COPD is one of more than 150 chronic conditions that are treatable with adult stem cells, eliminating the potential risk of surgery, transplants, and toxic drugs

Details of the protocol and eligibility criteria can be found on the government clinical trial website at: http://www.clinicaltrials.gov.

For more information on Global Stems Cell Group, visit the Global Stem Cells Group website, email bnovas(at)regenestem(dot)com, or call 305-224-1858.

About Global Stem Cells Group:

Global Stem Cells Group, Inc. is the parent company of six wholly owned operating companies dedicated entirely to stem cell research, training, products and solutions. Founded in 2012, the company combines dedicated researchers, physician and patient educators and solution providers with the shared goal of meeting the growing worldwide need for leading edge stem cell treatments and solutions.

With a singular focus on this exciting new area of medical research, Global Stem Cells Group and its subsidiaries are uniquely positioned to become global leaders in cellular medicine.

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Global Stem Cells Group, Inc. and BioHeart, Inc. Launch Clinical Trial for COPD Stem Cell Therapies

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TotipotentRX And ThermoGenesis Achieve Bone Marrow Stem …

RANCHO CORDOVA, Calif., LOS ANGELES and NEW DELHI, India, Feb. 3, 2014 (GLOBE NEWSWIRE) -- ThermoGenesis Corp. (Nasdaq:KOOL) a cellular therapy medical device company and TotipotentRX Corporation, a clinical-stage regenerative medicine company developing novel therapies for cardiovascular and orthopedic disease, announced the TotiPotentRX cellular therapy clinical team in partnership with Fortis Healthcare, Gurgaon (New Delhi) has achieved its 20 th pediatric bone marrow transplant (BMT). This haploidentical BMT was performed from a mother as a donor for a 10 year old child suffering from combined immunodeficiency due to a DOCK-8 gene mutation. The Fortis Centre has so far performed 15 allogenetic BMT including five haploidentical and one double unrelated cord blood transplants, and 5 autologous transplants. This transplant was completed on February 1, 2014 at the Pediatric Hematology and Bone Marrow Transplant department led by Dr. Satya Yadav, M.D., Head of the Department for Pediatric Hematology and Bone Marrow Transplant, and with scientific and laboratory support by the TotipotentRX's cell therapy GMP laboratory facility. This 20 th transplant is a significant milestone in the pursuit of developing the new FMRI BMT program into one of the leading stem cell transplant centers in Asia.

TotipotentRX provides laboratory services and scientific support to Fortis' cutting edge program at FMRI, some of which employs a proprietary approach to the transplant using the ThermoGenesis AutoXpress AXP and MarrowXpress MXP platforms when the processing of the donor's mobilized peripheral blood or bone marrow is required. These technologies allow for a proprietary transplant approach that increases pediatric patient access to this life saving treatment by enabling the following types of transplants that might otherwise not be an option for the patient:

Dr. Yadav remarked, "this 20 th transplant is a significant milestone for our patients, our research hospital and our transplant team.Achieving 15 allogenetic and 5 autologous transplants in the first half year of our program is remarkable for any leading academic institution.Our goal is to have the most advanced pediatric bone marrow transplant program in India, whilst taking a global leadership role in advanced therapy like the haploidentical transplant approach.We look forward to continuing our cutting edge program with TotipotentRX as a scientific collaborator."

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TotipotentRX And ThermoGenesis Achieve Bone Marrow Stem ...

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The Stem Cell Experts – Video


The Stem Cell Experts
The McGowan Institute for Regenerative Medicine is a program of the University of Pittsburgh and UPMC. The Institute specializes in discovering the potential...

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Innovative technique creates large skin flaps for full-face resurfacing

PUBLIC RELEASE DATE:

5-Feb-2014

Contact: Connie Hughes Connie.Hughes@wolterskluwer.com 646-674-6348 Wolters Kluwer Health

Philadelphia, Pa. (February 4, 2014) - Patients with massive burns causing complete loss of the facial skin pose a difficult challenge for reconstructive surgeons. Now a group of surgeons in China have developed an innovative technique for creating a one-piece skin flap large enough to perform full-face resurfacing, reports The Journal of Craniofacial Surgery, published by Lippincott Williams & Wilkins, a part of Wolters Kluwer Health.

Dr. QingFeng Li and colleagues of Shanghai Jiao Tong University School of Medicine describe their approach to creating "monoblock" flaps for use in extensive face skin resurfacing. In their successful experience with five severely disfigured patients, the full-face tissue flap "provides universally matched skin and near-normal facial contour."

New Technique Grows One-Piece Skin Flaps for Full-Face Resurfacing

Complete destruction of the facial skin and underlying (subcutaneous) tissues presents "the most challenging dilemma" in facial reconstructive surgery. Multiple skin flaps and grafts are needed to provide complete coverage, creating a "patchwork" appearance. Standard skin grafts are also too bulky to provide good reconstruction of the delicate features and expressive movement of the normal facial skin.

To meet these challenges, Dr. Li and colleagues have developed a new technique for creating a single, large skin flap appropriate for use in full-face resurfacing. Their approach starts with "prefabrication" of a flap of the patient's own skin, harvested from another part of the body. The skin flap, along with its carefully preserved blood supply, is allowed to grow for some weeks in a "pocket" created under the patient's skin of the patient's upper chest.

Tissue expandersballoon-like devices gradually filled with saline solutionare used to enlarge the skin flap over time. While skin expansion is a standard technique for creation of skin flaps, Dr. Li and his team used an "overexpansion" approach to create very large flaps of relatively thin skinideal for use in the facial area. In some cases, when the skin flap was growing too thin, stem cells derived from the patients' own bone marrow were used as an aid to tissue expansion.

Using this technique, Dr. Li and colleagues were able to create very large skin flapsup to 30 30 cmfor use in full-face resurfacing. In the new article, they describe their use of their prefabrication/overexpansion technique in five patients with complete loss of the facial skin, caused by flame or chemical burns. All patients had previously undergone facial reconstruction, but were left with severe deformity and limited facial movement.

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Innovative technique creates large skin flaps for full-face resurfacing

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therapy treatment for stem cell therapy treatment for cerebral palsy by dr alok sharma, mumbai, – Video


therapy treatment for stem cell therapy treatment for cerebral palsy by dr alok sharma, mumbai,
improvement seen in just 5 days after stem cell therapy treatment for cerebral palsy by dr alok sharma, mumbai, india. Stem Cell Therapy done date 31 Dec 201...

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14 Month Results After Stem Cell Therapy by Dr Harry Adelson for Arthritic Hip – Video


14 Month Results After Stem Cell Therapy by Dr Harry Adelson for Arthritic Hip
http://www.docereclinics 14 months after stem cell therapy for his arthritic hip, Marty discusses his results by Dr. Harry Adelson. Call the clinic today at ...

By: Harry Adelson, N.D.

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Webinar: Reimbursement Strategies for Stem Cell Therapy Products – Video


Webinar: Reimbursement Strategies for Stem Cell Therapy Products
Planning Your Payment Strategy in Early Product Development EXPERT SPEAKERS: Michael Werner, J.D., Executive Director, Alliance for Regenerative Medicine;...

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New stem cell research removes reliance on human and animal cells

13 hours ago

A new study, published today in the journal Applied Materials & Interfaces, has found a new method for growing human embryonic stem cells, that doesn't rely on supporting human or animal cells.

Traditionally, these stem cells are cultivated with the help of proteins from animals, which rules out use in the treatment of humans. Growing stem cells on other human cells risks contamination with pathogens that could transmit diseases to patients.

The team of scientists led by the University of Surrey and in collaboration with Professor Peter Donovan at the University of California have developed a scaffold of carbon nanotubes upon which human stem cells can be grown into a variety of tissues. These new building blocks mimic the surface of the body's natural support cells and act as scaffolding for stem cells to grow on. Cells that have previously relied on external living cells can now be grown safely in the laboratory, paving the way for revolutionary steps in replacing tissue after injury or disease.

Dr Alan Dalton, senior lecturer from the Department of Physics at the University of Surrey said: "While carbon nanotubes have been used in the field of biomedicine for some time, their use in human stem cell research has not previously been explored successfully."

"Synthetic stem cell scaffolding has the potential to change the lives of thousands of people, suffering from diseases such as Parkinson's, diabetes and heart disease, as well as vision and hearing loss. It could lead to cheaper transplant treatments and could potentially one day allow us to produce whole human organs without the need for donors."

Explore further: New method increases supply of embryonic stem cells

A new method allows for large-scale generation of human embryonic stem cells of high clinical quality. It also allows for production of such cells without destroying any human embryos. The discovery is a big step forward ...

A research team has identified a protein that increases the transfer of mitochondria from mesenchymal stem cells to lung cells. In work published in The EMBO Journal, the researchers reveal that the delivery of mitochondria to hum ...

Abba Zubair, M.D., Ph.D, believes that cells grown in the International Space Station (ISS) could help patients recover from a stroke, and that it may even be possible to generate human tissues and organs in space. He just ...

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New stem cell research removes reliance on human and animal cells

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Inner workings of a cellular nanomotor revealed

20 hours ago In this rendering of the SecA nanomotor, the two pistons (colored cyan and light green) await the arrival of a protein. Cylinders, arrows and loops make up the nanomotor's mechanical parts. Credit: T. Economou

Our cells produce thousands of proteins but more than one-third of these proteins can fulfill their function only after migrating to the outside of the cell. While it is known that protein migration occurs with the help of various 'nanomotors' that push proteins out of the cell, little is known about their precise mechanical functioning. New research by Anastassios Economou (KU Leuven) and his team reveals the inner workings of one such nanomotor, called SecA, with new clarity.

Protein migration is a fundamental problem in biology and is essential for life. Examples of migrating proteins include insulin (the absence of which leads to diabetes), antibodies (essential for combating infections), membrane channels (essential for neuronal cell function) and toxin-proteins (secreted by pathogenic microorganisms).

Migrating proteins contain chemical signals called signal peptides. These signal peptides act as postal addresses and direct exported proteins to the membrane for transport outside the cell.

In previous research, Dr Economou, in collaboration with Babis Kalodimos (Rutgers University), revealed how signal peptides bind to a specific cellular receptor on the membrane, which subsequently connects to the export channel leading out of the cell. This receptor was also found to act as a nanomotor, with two separate piston-like mechanical parts that somehow push proteins out of the cell. The exact mechanism by which this occurred remained a mystery. Until now.

In the current study, published in the December issue of Molecular Cell, Economou and his team show how the SecA receptor moves to push proteins out of the cell: when a signal peptide makes contact, the two pistons become excited. They position themselves one against the other in a series of defined steps and modify their shape. This finely orchestrated series of motions opens the export channel and traps the exported protein inside it. In a final step, the two parts dissociate and the remaining single piston pushes the protein out in cycles of repeated motions.

The discovery adds a significant piece in the puzzle of exploiting protein migration to improve health. "Moving forward, this discovery will help us focus efforts to find specific antibiotics against harmful bacterial protein secretion pathways," says Dr Economou. "It also offers the possibility to optimise biotechnological production of human biopharmaceuticals by using microbial 'cell factories' for secreting biopharmaceuticals. We will be pursuing these avenues in future research."

Explore further: Designer proteins provide new information about the body's signal processes

More information: The paper "Quaternary Dynamics of the SecA Motor Drive Translocase Catalysis" by Giorgos Gouridis, Spyridoula Karamanou, Marios Frantzeskos Sardis, Martin Alexander Schrer, Guido Capitani and Anastassios Economou can be accessed on the website of Molecular Cell: http://www.cell.com/molecular-cell/abstract/S1097-2765(13)00860-5

Journal reference: Molecular Cell

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Inner workings of a cellular nanomotor revealed

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Policymakers and scientists agree on top research questions

20 hours ago

Natural resource managers, policymakers and their advisers, and scientists have similar ideas about which research questions could, if answered, most increase the effectiveness of US natural resource management policies. A survey of over 600 members of those professional groups revealed how they ranked the importance of 40 management-relevant research questions identified in earlier work. Respondents judged the most important of the 40 questions to be the quantity and quality of surface and groundwater that will be necessary to sustain US human populations and ecosystem resilience during the next 100 years.

The Internet-based survey that yielded the rankings is described in an article to be published in the March issue of BioScience by Murray Rudd, of the University of York, United Kingdom, and Erica Fleishman, of the University of California, Davis.

The researchers used a technique called best-worst scaling to eliminate some of the biases that often confound rating studies: respondents repeatedly ranked small subsets of the 40 questions.

The questions had been identified earlier by natural resource managers, policymakers, and their advisers.

Statistical analysis of the survey results revealed the existence of subgroups with similar ideas about the importance of some questions, but there were no significant differences between policymakers and scientists, a result that surprised Rudd and Fleishman.

They are continuing to analyze results from this and a follow-up survey on related questions.

Explore further: The science of baby-making still a mystery for many women

More information: The earlier study identifying the 40 management-relevant research questions was published in BioScience in 2011 and can be read at bioscience.oxfordjournals.org/content/61/4/290.full

A wide-ranging group of experts has published a set of 40 key environmental questions to help align scientific research agendas with the needs of natural resource decision makers.

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Policymakers and scientists agree on top research questions

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Research and Markets: Concise Analysis of the …

DUBLIN--(BUSINESS WIRE)--Research and Markets (http://www.researchandmarkets.com/research/7pg67k/gene_therapy) has announced the addition of a new report "Concise Analysis of the International Gene Therapy Market: Updated 2014 Report" to their offering.

Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.

Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included.

Research and development is in progress in both the academic and the industrial sectors. The National Institutes of Health (NIH) of the US is playing an important part. As of 2013, over 2030 clinical trials have been completed, are ongoing or have been approved worldwide.A breakdown of these trials is shown according to the areas of application.

Since the death of Jesse Gelsinger in the US following a gene therapy treatment, the FDA has further tightened the regulatory control on gene therapy. A further setback was the reports of leukemia following use of retroviral vectors in successful gene therapy for adenosine deaminase deficiency.

Key Topics Covered:

Technologies & Markets

Executive Summary

1. Introduction

2. Gene Therapy Technologies

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