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.

Researchers study reproductive stage that remains most mysterious

Mississippi State University scientists are working to find out why some pregnancies are successful and others are not.

31 Aug 2013

Researchers identify mutation in CNGB1 gene causing PRA in Phalene and Papillon

Professor Hannes Lohi's research group at the University of Helsinki and Folkh-lsan Research Center, Finland, has identified a mutation in CNGB1 gene, causing progressive retinal atrophy (PRA) in the Phalene and Papillon dog breeds.

30 Aug 2013

Stem cell researchers receive Early Translational research awards from CIRM

Four prominent researchers from UCLA's Eli & Edythe Broad Center of Regenerative Medicine and Stem Cell Research have received Early Translational research awards totaling approximately $13 million from the California Institute for Regenerative Medicine (CIRM) the state stem cell agency. The UCLA researchers received four of the 12 total awards; no other applicant institution received more than one award.

30 Aug 2013

Scientists discover mechanisms of GATA3 gene that govern blood stem cell self-renewal process

Stem cell scientists at the Princess Margaret Cancer Centre have discovered the gene GATA3 has a role in how blood stem cells renew themselves, a finding that advances the quest to expand these cells in the lab for clinical use in bone marrow transplantation, a procedure that saves thousands of lives every year.

26 Aug 2013

New technique may convert cells from heart disease patients into heart muscle cells

Researchers have developed a new technique that might one day be used to convert cells from heart disease patients into heart muscle cells that could act as a personalized treatment for their condition.

23 Aug 2013

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

Gene Therapy News and Research

Further Reading

Researchers study reproductive stage that remains most mysterious

Researchers identify mutation in CNGB1 gene causing PRA in Phalene and Papillon

Stem cell researchers receive Early Translational research awards from CIRM

Scientists discover mechanisms of GATA3 gene that govern blood stem cell self-renewal process

New technique may convert cells from heart disease patients into heart muscle cells

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

Event guide: 9th Gene Therapy Development Summit 2026 eBook

The Critical Role of Sample Storage in Drug Discovery and Development

Advancing Neuroscience Research Through Collaboration and Patient-Centered Innovation

The impact of vibration on microscopy

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