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.

Gene therapy for rare genetic blindness wins prestigious Champalimaud Vision award

A team of seven researchers collaborating from United States and Britain have found a way to use gene therapy to cure a rare form of childhood blindness that occurs due to a genetic predisposition.

5 Sep 2018

Novel lentiviral model can determine effect of Wnt/β-Catenin inhibition on bone regeneration

Researchers have developed a novel tool for determining the sensitivity of bone healing to inhibition of the Wnt signaling pathway and have validated its use in a study of bone regeneration in mice.

4 Sep 2018

Stanford researchers show how to prevent immune response to gene therapy in mouse model

Stanford University School of Medicine researchers have demonstrated that gene therapy can be effective without causing a dangerous side effect common to all gene therapy: an autoimmune reaction to the normal protein, which the patient's immune system is encountering for the first time.

4 Sep 2018

Pfizer terminates clinical studies evaluating domagrozumab for treatment of Duchenne muscular dystrophy

Pfizer Inc. announced today that it is terminating two ongoing clinical studies evaluating domagrozumab (PF-06252616) for the treatment of Duchenne muscular dystrophy (DMD): a Phase 2 safety and efficacy study (B5161002) and an open-label extension study (B5161004).

30 Aug 2018

Using novel gene therapy to cure infants born with severe combined immunodeficiency

Out of every 60,000 births, a baby arrives to face the world without a fully functioning immune system leaving them unequipped to fight even the most common infections.

29 Aug 2018

Cherwell confirms support for conference addressing key challenges in aseptic manufacturing of ATMPs

Cherwell Laboratories, supplier of environmental monitoring and process validation products, has confirmed its continued support for the Pharmaceutical & Healthcare Sciences Society - UCL Q3P Annual Conference 2018.

29 Aug 2018

MDA announces 34 new grants totaling $9.9 million to accelerate neuromuscular research

The Muscular Dystrophy Association today announced the award of 34 new grants totaling more than $9.9 million for its summer round of funding. These new grants represent a continued commitment by MDA to fund groundbreaking research that will accelerate treatments and cures for the more than 40 diseases in its program.

24 Aug 2018

Bluebird bio collaborates with Gritstone Oncology to develop novel cancer cell therapies

Bluebird bio, Inc. and Gritstone Oncology, Inc. today announced a collaboration to research, develop and commercialize products for the treatment of cancer using cell therapy.

23 Aug 2018

Harvard scientists end 40-year-quest for elusive identity of 'hearing' protein

Scientists at Harvard Medical School say they have ended a 40-year-quest for the elusive identity of the sensor protein responsible for hearing and balance.

23 Aug 2018

Genetic variants impact outcome in dilated cardiomyopathy patients of African ancestry

Genetic testing is a powerful diagnostic tool that is increasingly being used for the diagnosis of dilated cardiomyopathy, a disease in which the heart becomes enlarged, making it difficult to pump blood. Cardiomyopathy affects more than 3.5 million people in the United States.

22 Aug 2018

Scientists publish study results of AAV therapy product for treatment of RHO-adRP

Ophthotech Corporation announced today that scientists at the University of Pennsylvania and University of Florida published proof-of-concept study results on an adeno-associated virus gene therapy product candidate for the treatment of rhodopsin-mediated autosomal dominant retinitis pigmentosa licensed by Ophthotech.

22 Aug 2018

Gene therapy with telomerase does not increase cancer risk, study shows

Negative results and findings in science are perhaps less newsworthy, but they are no less important. Particularly when, as in this case, they demonstrate that a possible new therapeutic pathway against idiopathic pulmonary fibrosis and other diseases associated to short telomeres is in fact safe.

21 Aug 2018

Scientists report novel gene therapy that halts vision loss in canine model of blinding disease

Scientists funded by the National Eye Institute report a novel gene therapy that halts vision loss in a canine model of a blinding condition called autosomal dominant retinitis pigmentosa (adRP). The strategy could one day be used to slow or prevent vision loss in people with the disease. NEI is part of the National Institutes of Health.

21 Aug 2018

Promising data supports development of AAVhu37-based gene therapy for hemophilia A

Gene therapy using an optimized adeno-associated virus to deliver the human factor VIII gene to cynomolgus macaques showed a substantial increase in hFVIII expression and no detectable antibody response for 30 weeks in some animals.

21 Aug 2018

New CRISPR technology may help eliminate mutated gene sequence

In a new study in cells, University of Illinois researchers have adapted CRISPR gene-editing technology to cause the cell's internal machinery to skip over a small portion of a gene when transcribing it into a template for protein building.

17 Aug 2018

Study shows efficacy, safety of AAV5-based gene therapy to treat sheep model of achromatopsia

A study of a large animal model of achromatopsia caused by a mutation in the CNGA3 gene that was treated with a single injection of CNGA3 gene therapy delivered using an AAV5 vector revealed findings reported long-term follow-up findings that show promise for the efficacy and safety of this therapeutic approach.

15 Aug 2018

Gene Therapy News and Research

Further Reading

Gene therapy for rare genetic blindness wins prestigious Champalimaud Vision award

Novel lentiviral model can determine effect of Wnt/β-Catenin inhibition on bone regeneration

Stanford researchers show how to prevent immune response to gene therapy in mouse model

Pfizer terminates clinical studies evaluating domagrozumab for treatment of Duchenne muscular dystrophy

Using novel gene therapy to cure infants born with severe combined immunodeficiency

Cherwell confirms support for conference addressing key challenges in aseptic manufacturing of ATMPs

MDA announces 34 new grants totaling $9.9 million to accelerate neuromuscular research

Bluebird bio collaborates with Gritstone Oncology to develop novel cancer cell therapies

Harvard scientists end 40-year-quest for elusive identity of 'hearing' protein

Genetic variants impact outcome in dilated cardiomyopathy patients of African ancestry

Scientists publish study results of AAV therapy product for treatment of RHO-adRP

Gene therapy with telomerase does not increase cancer risk, study shows

Scientists report novel gene therapy that halts vision loss in canine model of blinding disease

Promising data supports development of AAVhu37-based gene therapy for hemophilia A

New CRISPR technology may help eliminate mutated gene sequence

Study shows efficacy, safety of AAV5-based gene therapy to treat sheep model of achromatopsia

A class of proteins shown to be effective in reducing drug-seeking behaviors

New study focuses on promise of gene therapy for Amish nemaline myopathy

Pioneering research takes major step forward in the fight against cystic fibrosis

Breakthrough in stem cell research could lead to cure for cystic fibrosis

Leveraging the power of automation to boost research

Automating research - solutions and best practices

How can microdialysis benefit drug development

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