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.

Research suggests gene therapy approach may be feasible option to treat Rett Syndrome

The concept behind gene therapy is simple: deliver a healthy gene to compensate for one that is mutated. New research published today in the Journal of Neuroscience suggests this approach may eventually be a feasible option to treat Rett Syndrome, the most disabling of the autism spectrum disorders.

21 Aug 2013

Basic cancer research at Creighton yields promising results for diagnosing, treating asthma

What started as basic cancer research at Creighton University has yielded promising results for diagnosing and treating asthma - and the National Institutes of Health (NIH) agrees, investing nearly $1.5 million in Creighton to find out more.

17 Aug 2013

Study shows enhancer RNA molecules may open new avenues for gene therapy

A study investigating the function of the recently discovered enhancer RNA molecules may open new avenues for gene therapy. According to the study researchers, altering the production and function of these molecules could affect the expression of genes and, in consequence, possibly also the progression of various diseases.

14 Aug 2013

Discovery Transformation Program funds four projects for diabetes research

The Minnesota Partnership for Biotechnology and Medical Genomics announced four research projects selected for funding from the 2013 Discovery Transformation Grant Program.

13 Aug 2013

Rice researchers frame rules to design viral capsid proteins for gene therapy

Rice University researchers are making strides toward a set of rules to custom-design Lego-like viral capsid proteins for gene therapy.

13 Aug 2013

Learning "grammar" helps determine whether gene gets switched on or not

Researchers have probed deep into the cell's genome, beyond the basic genetic code, to begin learning the "grammar" that helps determine whether or not a gene gets switched on to make the protein it encodes.

13 Aug 2013

Gene combination therapy repairs and prevents future damage of muscles caused by DMD

New research on two promising gene therapies suggests that combining them into one treatment not only repairs muscle damage caused by Duchenne muscular dystrophy, but also prevents future injury from the muscle-wasting disease.

12 Aug 2013

Cognitive decline with age is natural part of life, OSU researchers find

If you forget where you put your car keys and you can't seem to remember things as well as you used to, the problem may well be with the GluN2B subunits in your NMDA receptors.

8 Aug 2013

Eteplirsen offers great hope to patients with Duchenne muscular dystrophy

Results from a clinical trial of eteplirsen, a drug designed to treat Duchenne muscular dystrophy, suggest that the therapy allows participants to walk farther than people treated with placebo and dramatically increases production of a protein vital to muscle growth and health.

8 Aug 2013

High-tech laser technique offers control of gene therapy and genetic engineering method

The applications of gene therapy and genetic engineering are broad: everything from pet fish that glow red to increased crop yields worldwide to cures for many of the diseases that plague humankind.

8 Aug 2013

UCLA researchers combine cellular and gene therapies to develop treatment for breast cancer

Researchers at UCLA's Jonsson Comprehensive Cancer Center have successfully combined cellular therapy and gene therapy in a mouse-model system to develop a viable treatment strategy for breast cancer that has spread to a patient's brain.

7 Aug 2013

TTY BIOPHARM receives Outstanding Company of the Year award

TTY BIOPHARM was awarded "Outstanding Company of the Year", the highest award given at the Bio Taiwan 2013 Exhibition. At the opening ceremony on July 18, Rong-Jing Lin, Chairman and General Manager of TTY BIOPHARM, received the prize from Ma Ying-jeou, President of Taiwan.

6 Aug 2013

Electronic communication unlikely to be widely adopted by physician practices

Patients like it and so do health organizations, but electronic communications in clinical care will likely not be widely adopted by primary care physicians unless patient workloads are reduced or they are paid for the time they spend phoning and emailing patients, both during and after office hours.

6 Aug 2013

Life Tech, Raintree Launch Oncology Testing, Patient Data Initiatives

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6 Aug 2013

BioLineRx reports positive results from pre-clinical trials with RHB-104 for treatment of thrombocytopenia

BioLineRx, a biopharmaceutical development company, announced today that BL-8040 has been shown in pre-clinical trials to be effective for the treatment of thrombocytopenia, or reduced platelet production.

5 Aug 2013

Study opens door to possible treatments for variety of mitochondrial diseases

By borrowing a tool from bacteria that infect plants, scientists have developed a new approach to eliminate mutated DNA inside mitochondria-the energy factories within cells. Doctors might someday use the approach to treat a variety of mitochondrial diseases, including the degenerative eye disease Leber hereditary optic neuropathy. The research, published online today in Nature Medicine, was funded by the National Eye Institute, a part of the National Institutes of Health.

5 Aug 2013

Trace substance in sugar improves muscle regeneration in mouse model with muscular dystrophy

A trace substance in caramelized sugar, when purified and given in appropriate doses, improves muscle regeneration in a mouse model of Duchenne muscular dystrophy. The findings are published today, Aug. 1, in the journal Skeletal Muscle.

2 Aug 2013

UCLA researchers develop treatment approach for breast cancer metastasized to brain

Researchers at UCLA's Jonsson Comprehensive Cancer Center (JCCC) have successfully combined cellular therapy and gene therapy in a mouse model system to develop a viable treatment strategy for breast cancer that has metastasized, or spread, to the patient's brain.

2 Aug 2013

Inhalable gene therapy may restore function of crucial enzyme to reverse deadly PAH

The deadly condition known as pulmonary arterial hypertension (PAH), which afflicts up to 150,000 Americans each year, may be reversible by using an inhalable gene therapy, report an international team of researchers led by investigators at the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai.

31 Jul 2013

Adaptimmune initiates Phase I/IIa open-label clinical trial in patients with ovarian cancer

Adaptimmune announced today that it has opened a Phase I/IIa, multiple-site, two-cohort, open-label clinical trial in ovarian cancer at Roswell Park Cancer Institute (RPCI) in Buffalo, N.Y., and City of Hope (COH) in Duarte, Calif.

26 Jul 2013

Gene Therapy News and Research

Further Reading

Research suggests gene therapy approach may be feasible option to treat Rett Syndrome

Basic cancer research at Creighton yields promising results for diagnosing, treating asthma

Study shows enhancer RNA molecules may open new avenues for gene therapy

Discovery Transformation Program funds four projects for diabetes research

Rice researchers frame rules to design viral capsid proteins for gene therapy

Learning "grammar" helps determine whether gene gets switched on or not

Gene combination therapy repairs and prevents future damage of muscles caused by DMD

Cognitive decline with age is natural part of life, OSU researchers find

Eteplirsen offers great hope to patients with Duchenne muscular dystrophy

High-tech laser technique offers control of gene therapy and genetic engineering method

UCLA researchers combine cellular and gene therapies to develop treatment for breast cancer

TTY BIOPHARM receives Outstanding Company of the Year award

Electronic communication unlikely to be widely adopted by physician practices

Life Tech, Raintree Launch Oncology Testing, Patient Data Initiatives

BioLineRx reports positive results from pre-clinical trials with RHB-104 for treatment of thrombocytopenia

Study opens door to possible treatments for variety of mitochondrial diseases

Trace substance in sugar improves muscle regeneration in mouse model with muscular dystrophy

UCLA researchers develop treatment approach for breast cancer metastasized to brain

Inhalable gene therapy may restore function of crucial enzyme to reverse deadly PAH

Adaptimmune initiates Phase I/IIa open-label clinical trial in patients with ovarian cancer

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