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.

Genetic variation linked to increased levels of triglycerides associated with heart disorders

Researchers at Weill Cornell Medical College have found that a genetic variation that is linked to increased levels of triglycerides -- fats in the blood associated with disorders such as heart disease, type 2 diabetes, obesity and stroke -- is far more common than previously believed and disproportionally affects people of African ancestry.

26 Nov 2013

Genetic diagnostic test for inherited eye diseases now available from Mass. Eye and Ear

The Massachusetts Eye and Ear/Harvard Medical School Department of Ophthalmology today announced the availability of a comprehensive genetic diagnostic test for inherited eye diseases.

22 Nov 2013

UK's first gene therapy trial for patients with chronic heart failure begins at GJNH

The UK’s first gene therapy trial for advanced heart failure, CUPID 2, has officially begun at the Golden Jubilee National Hospital; with the first candidate recently being administered with a dose of the MYDICAR treatment.

21 Nov 2013

Researchers report long-term benefits of gene therapy in advanced heart failure patients

Researchers from the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai reported the long-term benefits of a single dose of their gene therapy AAV1/SERCA2a in advanced heart failure patients on Nov. 19 at the American Heart Association Scientific Sessions 2013.

20 Nov 2013

Weill Cornell Medical College receives grant to support research efforts designed to end women's cancers

MasterCard today announced a $500,000 grant to Weill Cornell Medical College to support research efforts designed to end women's cancers.

19 Nov 2013

Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression

Successful gene therapy is based on the effective delivery and maintained expression of healthy copies of a gene into tissues of individuals with a disease-associated genetic mutation.

16 Nov 2013

Reengineered protein may lead to safer, more effective retroviral gene therapy

A fusion protein engineered by researchers at KU Leuven combining proteins active in HIV and Moloney murine leukaemia virus (MLV) replication may lead to safer, more effective retroviral gene therapy.

16 Nov 2013

Scientists join patients to address Friedreich's Ataxia

The Spanish Federation of Ataxia (FEDAES)—in representation of the GENEFA Platform for a Friedreich's Ataxia cure—, the Babel Family association for biomedical research into Friedreich's Ataxia, the "Centro de Biología Molecular Severo Ochoa" (CMBSO), and the Institute for Research in Biomedicine (IRB Barcelona) have signed an agreement through which these patients' associations will fund, by means of donations, a 3-year research project addressing Friedreich's Ataxia.

16 Nov 2013

Mount Sinai researchers successfully test SUMO-1 gene therapy to reverse heart failure

Researchers at the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai have successfully tested a powerful gene therapy, delivered directly into the heart, to reverse heart failure in large animal models.

14 Nov 2013

Professor receives NOK 75M grant to develop idea for next generation personalized cancer therapy

Extensive statistical analyses of the mutation distribution in several thousand cancerous tumours make it possible to find cures for types of cancer that cannot be treated today.

13 Nov 2013

Findings raise prospects for new therapies to protect heart from progressive disease

Some of the most commonly prescribed drugs for the treatment of heart failure are beta-blockers and nitrates, which help to relax blood vessels and decrease the heart's workload. The drugs were thought to produce those effects through distinct molecular pathways, but according to a new study led by scientists at Temple University School of Medicine, both types of drugs may help the failing heart by counteracting the effects of an enzyme known as GRK2.

8 Nov 2013

Michael Kors gifts $3M to Weill Cornell Medical College to support immunology research

Award-winning fashion designer Michael Kors has dedicated his career to designing luxury American sportswear. Now, with a $3 million gift to Weill Cornell Medical College, one of America's most recognized faces in fashion is seeking to significantly help advance human health.

8 Nov 2013

Researchers identify enzymes that are crucial for growth of cancers with genetic aberrations in p53

The most frequently mutated gene across all types of cancers is a gene called p53. Unfortunately it has been difficult to directly target this gene with drugs. Now a multi-institutional research team, led by Dr. Lewis Cantley and investigators at Weill Cornell Medical College, has identified a family of enzymes they say is crucial for the growth of cancers that have genetic aberrations in p53.

8 Nov 2013

Scientists uncover new mechanism through which gene expression is regulated

A new study by Weill Cornell Medical College scientists reveals a mechanism through which the expression of genes is controlled -- a finding that highlights genetic mutations that can impair the timing of gene expression.

31 Oct 2013

Researchers overcome obstacle to using viruses to deliver therapeutic genes

Scientists in The Research Institute at Nationwide Children's Hospital have found a way to overcome one of the biggest obstacles to using viruses to deliver therapeutic genes: how to keep the immune system from neutralizing the virus before it can deliver its genetic payload. In a study published recently in Molecular Therapy, researchers found that giving subjects a treatment to temporarily rid the body of antibodies provides the virus safe passage to targeted cells, allowing it to release a corrective or replacement gene to treat disease.

30 Oct 2013

State highlights: 'Habilitation' an essential benefit, but what is it?; Va. gov. candidates differ on mental health system; Calif. pays $2.7M for late hearings

A selection of health policy stories from Virginia, California, Colorado, Missouri, Pennsylvania, Kansas and Texas.

25 Oct 2013

Researchers use nanopores to smuggle DNA or proteins across membranes

A living cell is built with barriers to keep things out - and researchers are constantly trying to find ways to smuggle molecules in.‬ ‪Professor Giovanni Maglia (Biochemistry, Molecular and Structural Biology, KU Leuven) and his team have engineered a biological nanopore that acts as a selective revolving door through a cell's lipid membrane. The nanopore could potentially be used in gene therapy and targeted drug delivery.‬‬‬‬‬‬‬

24 Oct 2013

Research Institute receives Fast Track status for its gene therapy product for treatment of SMA

AveXis and BioLife, synthetic biology platform companies, today announced that The Research Institute at Nationwide Children's Hospital received Fast Track designation from the U.S. Food and Drug Administration for its scAAV9.CB.SMN gene therapy product for the treatment of spinal muscular atrophy (SMA).

19 Oct 2013

Researchers identify C. perfringens type B bacteria believed to trigger multiple sclerosis

A research team from Weill Cornell Medical College and The Rockefeller University has identified a bacterium it believes may trigger multiple sclerosis, a chronic, debilitating disorder that damages myelin forming cells in the brain and spinal cord.

18 Oct 2013

Detroit Lions, Josephine Ford Cancer Institute to raise $15M for cancer research in metro Detroit

The Detroit Lions and Josephine Ford Cancer Institute, Michigan's largest provider of adult cancer care, have created a unique partnership with the goal to raise $15 million during the next three years for cancer research in metro Detroit.

16 Oct 2013

Gene Therapy News and Research

Further Reading

Genetic variation linked to increased levels of triglycerides associated with heart disorders

Genetic diagnostic test for inherited eye diseases now available from Mass. Eye and Ear

UK's first gene therapy trial for patients with chronic heart failure begins at GJNH

Researchers report long-term benefits of gene therapy in advanced heart failure patients

Weill Cornell Medical College receives grant to support research efforts designed to end women's cancers

Human Treg responses allow sustained recombinant adeno-associated virus-mediated transgene expression

Reengineered protein may lead to safer, more effective retroviral gene therapy

Scientists join patients to address Friedreich's Ataxia

Mount Sinai researchers successfully test SUMO-1 gene therapy to reverse heart failure

Professor receives NOK 75M grant to develop idea for next generation personalized cancer therapy

Findings raise prospects for new therapies to protect heart from progressive disease

Michael Kors gifts $3M to Weill Cornell Medical College to support immunology research

Researchers identify enzymes that are crucial for growth of cancers with genetic aberrations in p53

Scientists uncover new mechanism through which gene expression is regulated

Researchers overcome obstacle to using viruses to deliver therapeutic genes

State highlights: 'Habilitation' an essential benefit, but what is it?; Va. gov. candidates differ on mental health system; Calif. pays $2.7M for late hearings

Researchers use nanopores to smuggle DNA or proteins across membranes

Research Institute receives Fast Track status for its gene therapy product for treatment of SMA

Researchers identify C. perfringens type B bacteria believed to trigger multiple sclerosis

Detroit Lions, Josephine Ford Cancer Institute to raise $15M for cancer research in metro Detroit

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