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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.
Society for Reproductive Investigation awards two top honors to Dr. Ayman Al-Hendy

Society for Reproductive Investigation awards two top honors to Dr. Ayman Al-Hendy

Dr. Ayman Al-Hendy, an obstetrician-gynecologist and molecular biologist at the Medical College of Georgia at Georgia Regents University and GRHealth, has received two top honors from the Society for Reproductive Investigation. [More]
MDC scientists find solution to increase efficiency of precise genetic modifications

MDC scientists find solution to increase efficiency of precise genetic modifications

CRISPR-Cas9 is a powerful new tool for editing the genome. For researchers around the world, the CRISPR-Cas9 technique is an exciting innovation because it is faster and cheaper than previous methods. Now, using a molecular trick, Dr. Van Trung Chu and Professor Klaus Rajewsky of the Max Delbrück Center for Molecular Medicine Berlin-Buch and Dr. Ralf Kühn, MDC and Berlin Institute of Health, have found a solution to considerably increase the efficiency of precise genetic modifications by up to eightfold. [More]
Novasep, Celladon sign agreement to develop and supply MYDICAR drug substance

Novasep, Celladon sign agreement to develop and supply MYDICAR drug substance

Novasep, a leading supplier of services and technologies for the life sciences and chemical industries, and Celladon Corporation, a clinical-stage cardiovascular gene therapy company, today announce that they have signed a Development, Manufacturing and Supply Agreement pursuant to which, if supported by upcoming MYDICAR clinical data, Novasep would manufacture MYDICAR drug substance through 2018 with extension options through 2020. [More]
Personalized gene therapy to fight against cancer

Personalized gene therapy to fight against cancer

The fight to treat cancer and eradicate tumors will likely benefit from a new set of treatments if early development phases continue to show promise, according to Kalorama Information. The healthcare market research publisher stated that gene therapies that are able to deliver genetic material to a specific cell population or tumor that will result in the destruction of the tumor. [More]
Experimental drug that attacks brain tumor cells passes early tests

Experimental drug that attacks brain tumor cells passes early tests

An experimental drug that attacks brain tumor tissue by crippling the cells' energy source called the mitochondria has passed early tests in animal models and human tissue cultures, say Houston Methodist scientists. [More]
Stempeutics' Stempeucel drug receives ATMP classification from EMA for treatment of TAO

Stempeutics' Stempeucel drug receives ATMP classification from EMA for treatment of TAO

Stempeutics Research, a group company of Manipal Education and Medical Group and a Joint Venture with Cipla Group, announced today that the European Medicines Agency has granted Advanced Therapy Medicinal Product classification for its novel stem cell drug 'Stempeucel' which will be used for the treatment of Thromboangiitis Obliterans (TAO). [More]
Researchers find key step in understanding genetic mechanism of plants' environmental adaptability

Researchers find key step in understanding genetic mechanism of plants' environmental adaptability

A fundamental question pursued by plant scientists worldwide for the past decade has been answered by researchers led by the University of Sydney in Australia. [More]
Researchers identify new gene variants that could contribute to sporadic motor neurone disease

Researchers identify new gene variants that could contribute to sporadic motor neurone disease

Researchers have identified a new host of gene variants that could make people vulnerable to sporadic motor neurone disease, according to a report published today in the journal, Scientific Reports. [More]
'Turbocharged' protein holds promise in hemophilia gene therapy

'Turbocharged' protein holds promise in hemophilia gene therapy

Using gene therapy to produce a mutant human protein with unusually high blood-clotting power, scientists have successfully treated dogs with the bleeding disorder hemophilia, without triggering an unwanted immune response. [More]
Study: New gene therapy safe, effective for patients with hemophilia B

Study: New gene therapy safe, effective for patients with hemophilia B

A multi-year, ongoing study suggests that a new kind of gene therapy for hemophilia B could be safe and effective for human patients. Published in the journal Science Translational Medicine, the research showed that a reprogrammed retrovirus could successfully transfer new factor IX (clotting) genes into animals with hemophilia B to dramatically decrease spontaneous bleeding. [More]
Finding could lead to more effective, less invasive treatment for 'bubble boy' disease

Finding could lead to more effective, less invasive treatment for 'bubble boy' disease

For infants with severe combined immunodeficiency (SCID), something as simple as a common cold or ear infection can be fatal. Born with an incomplete immune system, kids who have SCID--also known as "bubble boy" or "bubble baby" disease--can't fight off even the mildest of germs. [More]
Fourth patient dosed in Benitec Biopharma's Phase I/IIa hepatitis C trial

Fourth patient dosed in Benitec Biopharma's Phase I/IIa hepatitis C trial

Benitec Biopharma, a biopharmaceutical company focused on providing potentially curative therapies with its proprietary gene-silencing technology called ddRNAi or "expressed RNAi," today announced that the fourth patient in the company's Phase I/IIa dose escalation clinical trial of its lead program TT-034 for treating hepatitis C was dosed at the Duke Clinical Research Unit. [More]
New drug shows promise in driving insulin-producing beta cells to multiply

New drug shows promise in driving insulin-producing beta cells to multiply

In a screen of more than 100,000 potential drugs, only one, harmine, drove human insulin-producing beta cells to multiply, according to a study led by researchers at the Icahn School of Medicine at Mount Sinai, funded by JDRF and the National Institutes of Health, and published online today in Nature Medicine. [More]
Viagra can have anti-cancer, anti-Alzheimer's disease effects if used with new drugs

Viagra can have anti-cancer, anti-Alzheimer's disease effects if used with new drugs

Chaperone proteins play an important role in protein folding in human cells and in bacteria and are promising new targets for drugs to treat cancer and Alzheimer's disease and for novel antiviral drugs and antibiotics. How existing drugs such as Viagra or Cialis and a derivative of the drug Celebrex, for example, can reduce the activity of a specific chaperone protein, with the potential for anti-tumor and anti-Alzheimer's disease effects, is described in a Review article in DNA and Cell Biology, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. [More]
Non-viral gene therapy provides relief for diabetics with constant foot pain

Non-viral gene therapy provides relief for diabetics with constant foot pain

Walking barefoot on sand "felt like walking on glass" for Keith Wenckowski, who has lived with type-one diabetes for more than two decades. [More]
MIT researchers develop new nanodevice that can help overcome drug resistance

MIT researchers develop new nanodevice that can help overcome drug resistance

Chemotherapy often shrinks tumors at first, but as cancer cells become resistant to drug treatment, tumors can grow back. A new nanodevice developed by MIT researchers can help overcome that by first blocking the gene that confers drug resistance, then launching a new chemotherapy attack against the disarmed tumors. [More]
New strategy may ensure safety of adult epidermal stem cells before performing treatments

New strategy may ensure safety of adult epidermal stem cells before performing treatments

A team of European researchers has devised a strategy to ensure that adult epidermal stem cells are safe before they are used as treatments for patients. The approach involves a clonal strategy where stem cells are collected and cultivated, genetically modified and single cells isolated before being rigorously tested to make sure they meet the highest possible safety criteria. [More]
RowanSOM researcher awarded NINDS grant to develop stem cell-based therapy for Canavan disease

RowanSOM researcher awarded NINDS grant to develop stem cell-based therapy for Canavan disease

Paola Leone, PhD, the director of the Cell and Gene Therapy Center and a professor of Cell Biology at the Rowan University School of Osteopathic Medicine, has been awarded a three-year, $477,000 grant from the National Institute of Neurological Disorders and Stroke to develop a stem cell-based therapy for Canavan disease, a rare but devastating neurological disorder in children that typically takes a child's life by age 10. [More]
Scientists discover DNA vaccine that alleviates chronic inflammation in the body

Scientists discover DNA vaccine that alleviates chronic inflammation in the body

An international team of scientists including CureLab Oncology, Inc. (Boston), University of Camerino (Italy), and Boston University have serendipitously discovered a DNA vaccine, which systemically alleviates chronic inflammation in the body. Since osteoporosis is an inflammatory disease, preventive and therapeutic effects of the new vaccine were demonstrated on mouse models with osteoporosis. [More]
New TAU study may offer hope to people diagnosed with Glioblastoma multiforme

New TAU study may offer hope to people diagnosed with Glioblastoma multiforme

There are no effective available treatments for sufferers of Glioblastoma multiforme (GBM), the most aggressive and devastating form of brain tumor. The disease, always fatal, has a survival rate of only 6-18 months. [More]
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