Gene Therapy News and Research

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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.

Biological scaffold for heart valves can be freeze-dried for later use

The biological scaffold that gives structure to a heart valve after its cellular material has been removed can be freeze-dried and stored for later use as a tissue-engineered replacement valve to treat a failing heart, as described in an article in Tissue Engineering, Part C: Methods, a peer-reviewed journal from Mary Ann Liebert, Inc..

15 Feb 2012

Machine-learning methods show that patients with severe brain injury could communicate accurately

Only by employing complex machine-learning techniques to decipher repeated advanced brain scans were researchers at NewYork-Presbyterian/Weill Cornell able to provide evidence that a patient with a severe brain injury could, in her way, communicate accurately.

14 Feb 2012

KSU, St. Michael's receive U.S. patent to use BRCA1 gene for cardiovascular disease

St. Michael's Hospital and King Saud University have received their first joint U.S. patent to use the BRCA1 gene as a therapy for cardiovascular disease.

13 Feb 2012

Gene therapy to promote remyelination in mouse model of MS

Our bodies are full of tiny superheroes-antibodies that fight foreign invaders, cells that regenerate, and structures that ensure our systems run smoothly. One such structure is myelin-a material that forms a protective, insulating cape around the axons of our nerve cells so that they can send signals quickly and efficiently. But myelin, and the specialized cells called oligodendrocytes that make it, become damaged in demyelinating diseases like multiple sclerosis (MS), leaving neurons without their myelin sheaths.

10 Feb 2012

New cERD method can detect RNAi activity in biological fluids

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that its scientists have discovered a novel method for monitoring RNAi activity in blood samples, representing a potential major advance for future development of RNAi therapeutics, other therapeutic modalities, and diagnostics.

10 Feb 2012

Gene therapy succeeds in the second blind eye for three patients

Three people have now have reported a dramatic improvement in vision after having gene therapy in both eyes after having lost their eyesight as children.

9 Feb 2012

Readministration of gene therapy improves vision in three patients with LCA

Gene therapy for congenital blindness has taken another step forward, as researchers further improved vision in three adult patients previously treated in one eye.

9 Feb 2012

Mary Ann Liebert to launch BioResearch Open Access in March

BioResearch Open Access, a new bimonthly peer-reviewed open access journal, will launch in March 2012 by Mary Ann Liebert, Inc., publishers.

9 Feb 2012

Tennessee Gov. declares February as 'St. Jude Month'

In 1962, St. Jude Children's Research Hospital opened its doors amid an emotionally charged debate regarding how to treat childhood cancer. At that time, few children with the most common form of childhood cancer survived, and many physicians believed treatment was futile. St. Jude physicians and researchers took a radically different approach, and these efforts proved pivotal in changing the way the world treats childhood cancer.

6 Feb 2012

Whole-exome sequencing discovers cause of glycosylation disorders

Sequencing a patient's entire genome to discover the source of his or her disease is not routine - yet. But geneticists are getting close. A case report, published this week in the American Journal of Human Genetics, shows how researchers can combine a simple blood test with an "executive summary" scan of the genome to diagnose a type of severe metabolic disease.

4 Feb 2012

Scientists discover new mechanisms by which RNA drugs can control gene activity

Small RNA-based nucleic acid drugs represent a promising new class of therapeutic agents for silencing abnormal or overactive disease-causing genes, and researchers have discovered new mechanisms by which RNA drugs can control gene activity.

3 Feb 2012

Weill Cornell establishes Office of Faculty Development

Weill Cornell Medical College has announced the establishment of the Office of Faculty Development dedicated to assisting its physicians, researchers and educators in achieving academic success at each stage of their careers, with targeted activities that focus on the needs of junior, mid-career and senior faculty.

27 Jan 2012

Creighton researcher to study role of adult stem cells in repairing damaged coronary arteries

A Creighton University School of Medicine researcher has received a $3.3 million, five-year grant from the National Institutes of Health to study what role adult stem cells might play in repairing damaged coronary arteries, a complication that often occurs in patients after they undergo angioplasty and stenting.

24 Jan 2012

Positive affect, self-affirmation help patients with chronic diseases to overcome obstacles

The experience of daily positive affect -- a mild, happy feeling -- and self-affirmation helps some patients with chronic diseases, including coronary artery disease, high blood pressure and asthma, make better decisions about their health.

24 Jan 2012

New gene therapy shows promise against X-linked retinitis pigmentosa

A new gene therapy method developed by University of Florida researchers has the potential to treat a common form of blindness that strikes both youngsters and adults.

24 Jan 2012

MSC-mediated RNAi transfer holds promise for Huntington's disease

A team of researchers at the UC Davis Institute for Regenerative Cures has developed a technique for using stem cells to deliver therapy that specifically targets the genetic abnormality found in Huntington's disease, a hereditary brain disorder that causes progressive uncontrolled movements, dementia and death.

20 Jan 2012

Aspirin may benefit HIV-infected women at risk of cervical cancer

Research conducted by NewYork-Presbyterian Hospital/Weill Cornell Medical Center global health investigators and cancer specialists in New York, Qatar and Haiti suggests that aspirin should be evaluated for its ability to prevent development of cervical cancer in HIV-infected women.

19 Jan 2012

USPTO issues new patent to PBL's RNAi technology

Plant Bioscience Limited (PBL) is pleased to announce that the United States Patent and Trademark Office (USPTO) has issued out US Patent No. 8,097,710 with fundamental claims directed to methods of inducing gene silencing using short RNA molecules, or DNA constructs encoding short RNA molecules, in a wide range of organisms, including in plants and humans.

19 Jan 2012

DBS safe and effective for patients with advanced Parkinson's

Researchers from the University of Florida and 14 additional medical centers reported results today in the online version of The Lancet Neurology journal indicating that deep brain stimulation - also known as DBS - is effective at improving motor symptoms and quality of life in patients with advanced Parkinson's disease.

12 Jan 2012

Bladder cancer is one of the top 10 most common forms of cancer in the US

Bladder cancer, most frequently caused by smoking and exposure to carcinogens in the workplace, is one of the top 10 most common forms of cancer in men and women in the U.S. More than 70 percent of bladder cancers are diagnosed in stage T1 or less and have not invaded the muscle layer.

11 Jan 2012