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.

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

Manchester scientists treat fatal genetic brain disease using stem cell gene therapy

Scientists from The University of Manchester have used stem cell gene therapy to treat a fatal genetic brain disease in mice for the first time.

From The University of Manchester 25 Jul 2013

Investigators report complete catalog of genes expressed in retina

Investigators at Massachusetts Eye and Ear and Harvard Medical School have published the most thorough description of gene expression in the human retina reported to date. In a study published today in the journal BMC Genomics, Drs. Michael Farkas, Eric Pierce and colleagues in the Ocular Genomics Institute at Mass. Eye and Ear reported a complete catalog of the genes expressed in the retina.

20 Jul 2013

Researchers identify cellular channels critical for hearing

Ending a 30-year search by scientists, researchers at Boston Children's Hospital have identified two proteins in the inner ear that are critical for hearing, which, when damaged by genetic mutations, cause a form of delayed, progressive hearing loss. Findings were published online July 18 by the journal Neuron.

19 Jul 2013

Audentes Therapeutics closes $30 million Series A financing

Audentes Therapeutics, Inc., a recently founded biotechnology company dedicated to the development of innovative treatments for rare muscle diseases, today announced the closing of a $30 million Series A financing.

18 Jul 2013

CHOP-led research increases numbers of patients who could be treated with gene therapy

Gene therapy researchers have produced a bioengineered decoy that fools the immune system and prevents it from mistakenly defeating the benefits delivered by a corrective gene.

18 Jul 2013

Scientists find way to sneak nanoparticles carrying anticancer drugs past cells of immune system

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17 Jul 2013

Smokers who seem healthy have damaged airway cells similar to cells found in aggressive lung cancer

Smokers who've received a clean bill of health from their doctor may believe cigarettes haven't harmed their lungs. However, researchers at Weill Cornell Medical College have found that even smokers who seem healthy have damaged airway cells, with characteristics similar to cells found in aggressive lung cancer.

17 Jul 2013

Weill Cornell reports new findings from IVIG Phase 3 trial in Alzheimer's disease at AAIC 2013

Weill Cornell Medical College neurologist Dr. Norman Relkin reported new findings today from the Phase 3 clinical trial of IVIG (intravenous immunoglobulin) in mild to moderate Alzheimer's disease at the Alzheimer's Association International Conference in Boston, Mass.

16 Jul 2013

'Defanged' HIV used to deliver gene therapy

Researchers are having success using a "defanged" version of HIV to deliver gene therapy to children suffering from genetic diseases, a development that could improve other such care.

15 Jul 2013

First Edition: July 12, 2013

Today's early morning highlights from the major news organizations, including articles on data tracking of doctors and comparing the rollout of the Medicare Part D drug plans to implementation of the federal health law.

12 Jul 2013

Hydrogel method form synthetic scaffolds to support formation of blood vessels in engineered tissues

Next-generation hydrogels can form synthetic scaffolds to support the formation of replacement tissues and organs in the emerging area of regenerative medicine. Embedding peptides into the hydrogels stimulates the growth of essential microvascular networks to ensure a good blood supply.

12 Jul 2013

Research on activating Glial Cells to be presented at 7th Annual Pain Therapeutics Summit East

Linda Watkins, Ph.D., Distinguished Professor, Department of Psychology & Neuroscience and Center for Neuroscience, University of Colorado, Co-Chair, Advisory Board, Xalud Therapeutics, Inc. will present research on activating Glial Cells as part of the speaking faculty at the 7th Annual Pain Therapeutics Summit East in Boston, MA.

11 Jul 2013

Action Medical Research funds three new Research Training Fellowships

Action Medical Research, the UK-wide charity funding vital research to help sick and disabled babies and children, has today announced it is funding three new Research Training Fellowships worth more than £470,000.

9 Jul 2013

3-gene signature test will improve care of kidney transplant patients

A breakthrough non-invasive test can detect whether transplanted kidneys are in the process of being rejected, as well as identify patients at risk for rejection weeks to months before they show symptoms, according to a study published in The New England Journal of Medicine (NEJM).

4 Jul 2013

Gene therapy developed by UAB cures pediatric Sanfilippo Syndrome A in animal models

A single session of a gene therapy developed by the Universitat Autònoma de Barcelona (UAB) cures Sanfilippo Syndrome A in animal models. This syndrome is a neurodegenerative disease that affects between 1 and 9 out of every 100,000 children, and causes the death of the child on reaching adolescence.

3 Jul 2013