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.

Nationwide Insurance Foundation donates $10M to Nationwide Children's Hospital

Nationwide Children's Hospital today announced that it has received a $10 million gift from the Nationwide Insurance Foundation.

27 May 2014

Study: Sufficient copies of SMN1 gene extend survival in animals with spinal muscular atrophy

To make up for insufficient amounts of SMN protein, the cause of the inherited neuromuscular disease spinal muscular atrophy (SMA), researchers have successfully delivered a replacement SMN1 gene directly to the spinal cords of animal models of SMA.

23 May 2014

Study: RNAi silencing strategy blocks production of mutant huntingtin protein

A targeted gene silencing strategy blocks production of the dysfunctional huntingtin (Htt) protein, the cause of Huntington's disease, a fatal, inherited neurodegenerative disorder.

22 May 2014

DNA-directed RNA interference and hepatitis C: an interview with Carl Stubbings, Chief Business Officer, Benitec Biopharma

RNA interference (RNAi) is a naturally occurring process, which cells can use to silence, or ‘turn off’ unwanted genes.

From Quest Diagnostics 19 May 2014

MD Anderson Cancer Center, Concord Medical sign consulting agreement for cancer hospital projects

Concord Medical Services Holdings Limited ("Concord Medical" or the "Company"), a leading specialty hospital management solution provider and operator of the largest network of radiotherapy and diagnostic imaging centers in China, today announced it has engaged The University of Texas MD Anderson Cancer Center ("MD Anderson") as a consultant for its cancer hospital projects under planning in Shanghai and Beijing.

16 May 2014

Researchers develop new high-tech device for transferring DNA into cells

The ability to transfer a gene or DNA sequence from one animal into the genome of another plays a critical role in a wide range of medical research-including cancer, Alzheimer's disease, and diabetes.

14 May 2014

Abeona Therapeutics obtains FDA Orphan Drug Designations for Sanfilippo Syndrome therapies

Abeona Therapeutics, a start-up company created around intellectual property licensed from Nationwide Children's Hospital to develop treatments for Sanfilippo Syndrome Types A and B, has been granted Orphan Drug Designations for its lead investigational therapies by the U.S. Food and Drug Administration Office of Orphan Products Development.

12 May 2014

Researchers reveal how protein acts as brake against pathological growth of heart muscle

Cardiovascular disease remains the number one cause of death in the Western world, with heart failure representing the fastest-growing subclass over the past decade.

12 May 2014

Early gene therapy could improve choroideremia prognosis

Retinal gene therapy could prevent loss of vision in patients with choroideremia and other retinal diseases, show the results of a phase I/II study.

30 Apr 2014

Researchers create tiny, biodegradable nanoparticles that carry DNA to brain cancer cells

Working together, Johns Hopkins biomedical engineers and neurosurgeons report that they have created tiny, biodegradable “nanoparticles” able to carry DNA to brain cancer cells in mice.

30 Apr 2014

Promising new approach targets cancer protein to slow tumor growth

Sometimes a full-on assault isn't the best approach when dealing with a powerful enemy. A more effective approach, in the long run, may be to target the support system replenishing the supplies that keep your foe strong and ready for battle.

26 Apr 2014

Researchers develop gene therapy reversing memory loss in mice with early-stage Alzheimer's

Alzheimer's disease is the first cause of dementia and affects some 400,000 people in Spain alone. However, no effective cure has yet been found. One of the reasons for this is the lack of knowledge on the cellular mechanisms which cause alterations in nerve transmissions and the loss of memory in the initial stages of the disease.

25 Apr 2014

Researchers devise new approach to treatment of Alzheimer's disease

A team of researchers from Columbia University Medical Center, Weill Cornell Medical College, and Brandeis University has devised a wholly new approach to the treatment of Alzheimer's disease involving the so-called retromer protein complex. Retromer plays a vital role in neurons, steering amyloid precursor protein (APP) away from a region of the cell where APP is cleaved, creating the potentially toxic byproduct amyloid-beta, which is thought to contribute to the development of Alzheimer's.

21 Apr 2014

Researchers discover new marker derived from human umbilical cord blood

The development of stem cell therapies to cure a variety of diseases depends on the ability to characterize stem cell populations based on cell surface markers.

18 Apr 2014

European scientists set up new therapeutic approaches to tackle gene defects

On 15th April is the 1st International Pompe Disease Day, a campaign to raise awareness of this rare but severe gene defect. Pompe Disease is only one of more than 40 metabolic disorders that mainly affect children under the age of 10, often with devastating consequences.

15 Apr 2014

Inserm plays key role in developing European research

The French National Institute of Health and Medical Research (Inserm) is currently the leading biomedical research organisation in Europe, and plays a key role in developing European research. To mark its 50th anniversary, Inserm wanted to assess the perceptions of Europeans regarding biomedical research.

10 Apr 2014

Study explores relationship between genetics and lifestyle in development of AD

A global study involving more than one million people worldwide will explore the relationship between genetics and lifestyle in the development of Alzheimer's disease.

4 Apr 2014

Expert in gene therapy joins The Children's Hospital of Philadelphia

Beverly L. Davidson, Ph.D., a nationally prominent expert in gene therapy, is joining The Children's Hospital of Philadelphia (CHOP) today.

2 Apr 2014

Cedars-Sinai earns grant to conduct clinical trial of gene therapy product for patients with Lou Gehrig's disease

The Cedars-Sinai Regenerative Medicine Institute has received a $2.5 million grant from the Department of Defense to conduct animal studies that, if successful, could provide the basis for a clinical trial of a gene therapy product for patients with Lou Gehrig's disease, also called amyotrophic lateral sclerosis, or ALS.

2 Apr 2014

Study points to potential culprit that kills motor neurons in ALS

Amyotrophic lateral sclerosis, also known as Lou Gehrig's disease, is marked by a cascade of cellular and inflammatory events that weakens and kills vital motor neurons in the brain and spinal cord. The process is complex, involving cells that ordinarily protect the neurons from harm. Now, a new study by scientists in The Research Institute at Nationwide Children's Hospital points to a potential culprit in this good-cell-gone-bad scenario, a key step toward the ultimate goal of developing a treatment.

27 Mar 2014

Gene Therapy News and Research

Further Reading

Nationwide Insurance Foundation donates $10M to Nationwide Children's Hospital

Study: Sufficient copies of SMN1 gene extend survival in animals with spinal muscular atrophy

Study: RNAi silencing strategy blocks production of mutant huntingtin protein

DNA-directed RNA interference and hepatitis C: an interview with Carl Stubbings, Chief Business Officer, Benitec Biopharma

MD Anderson Cancer Center, Concord Medical sign consulting agreement for cancer hospital projects

Researchers develop new high-tech device for transferring DNA into cells

Abeona Therapeutics obtains FDA Orphan Drug Designations for Sanfilippo Syndrome therapies

Researchers reveal how protein acts as brake against pathological growth of heart muscle

Early gene therapy could improve choroideremia prognosis

Researchers create tiny, biodegradable nanoparticles that carry DNA to brain cancer cells

Promising new approach targets cancer protein to slow tumor growth

Researchers develop gene therapy reversing memory loss in mice with early-stage Alzheimer's

Researchers devise new approach to treatment of Alzheimer's disease

Researchers discover new marker derived from human umbilical cord blood

European scientists set up new therapeutic approaches to tackle gene defects

Inserm plays key role in developing European research

Study explores relationship between genetics and lifestyle in development of AD

Expert in gene therapy joins The Children's Hospital of Philadelphia

Cedars-Sinai earns grant to conduct clinical trial of gene therapy product for patients with Lou Gehrig's disease

Study points to potential culprit that kills motor neurons in ALS

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