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Gene therapy gives clue to treatment

Thu, 10/07/2010 - 10:29 by Rakhi

Chapel Hill, N.C. -- A first clinical trial to replace a genetic defect in patients with muscular dystrophy has yielded an unexpected and encouraging result, U.S. researchers say.

University of North Carolina at Chapel Hill School of Medicine researchers have made a discovery about a muscle protein called dystrophin, an essential protein that is deficient in those suffering the disease, a university release said.

The illness occurs when a gene on the X chromosome fails to make the vital protein. In the gene therapy trial it was found that the immune system of muscular dystrophy patients, once thought to be devoid of dystrophin, is "primed" by the prior existence of a tiny amount of the vital protein that escaped the effects of the mutation.

Gene therapy used in retinitis pigmentosa

Wed, 06/16/2010 - 10:43 by Rakhi

Oklahoma City -- U.S. medical investigators say they have developed a gene therapy technique that can prevent and possibly cure blindness caused by retinitis pigmentosa.

University of Oklahoma Health Sciences Center researchers said they found a way to use a radical new type of gene therapy that gives hope to the estimated 100,000 U.S. citizens who suffer from retinitis pigmentosa -- an eye disease in which genetic damage to the retina gradually becomes worse and can eventually lead to blindness.

Gene therapy cures dogs of color blindness

Fri, 04/23/2010 - 12:04 by Rakhi

Philadelphia -- University of Pennsylvania scientists say they have used gene therapy to restore retinal cone function and day vision in two canine models of color blindness.

The veterinary ophthalmology researchers said the disorder -- called congenital achromatopsia or rod monochromacy -- is a rare autosomal recessive genetic disorder with an estimated prevalence in human beings of about 1 in 50,000.

Cone function is essential for color vision, central visual acuity and most daily visual activities, which underlines the importance of the newly developed treatment, the scientists said.

Gene therapy useful in emphysema

Wed, 12/23/2009 - 07:03 by harsheeb

Boston -- A new gene therapy involving a single treatment could prevent the progression of inherited emphysema, scientists in Boston said.

Using mice, researchers at Boston University School of Medicine prevented the progression of Alpha-1 Anti-trypsin Deficiency, the most common inherited form of emphysema seen in young people, the university said in release. People who inherit the deficiency are predisposed to early emphysema and cirrhosis of the liver.

The researchers were able to deliver therapeutic genes to as much as 70 percent of a mouse lung's alveolar macrophages, a cell type that contributes to emphysema.

Gene therapy useful in stabilizing Lorenzo's Oil disease

Sun, 11/08/2009 - 15:52 by Jaspreet Kaur

Paris, November 8 -- French researchers have achieved a rare feat of stopping the progression of an uncommon and deadly degenerative disorder called adrenoleukodystrophy (ALD) in two boys by using gene therapy.

Scientists repair damaged lungs for transplantation

Thu, 10/29/2009 - 17:00 by Jaspreet Kaur

Washington, October 29 -- As per a latest study published Wednesday, damaged lungs can finally be repaired by performing a genetic patch job. Researchers from Canada fixed up worn out lungs that had been donated, using outside-the-body gene therapy.

Gene therapy restores partial vision

Sun, 10/25/2009 - 14:23 by Neka Sehgal

Philadelphia, October 24 -- There is heartening news for those who are virtually blind due to a genetically-inherited sight disorder. Doctors in the U.S. have been successful in restoring some sight in people with a previously incurable eye condition known as Leber’s congenital amaurosis (LCA) by using an experimental form of gene therapy.

Gene injection gives color-blind monkeys normal color vision

Thu, 09/17/2009 - 11:14 by Camilla Abad

September 17, Seattle: Color blind individuals may now have a chance to achieve normal vision after an experiment involving gene therapy cured two color-blind monkeys.

Therapy cures colorblindness in monkeys

Thu, 09/17/2009 - 01:39 by surajdogra

Gainesville, Fla. -- U.S. scientists say gene therapy used to cure colorblindness in two squirrel monkeys might lead to similar treatments to cure adult human vision disorders.

Researchers from the University of Florida and the University of Washington said their new gene therapy has the potential to treat human disorders involving cone cells in the eyes. Colorblindness, they noted, is the most common genetic disorder occurring in humans

New method of gene therapy is developed

Wed, 05/06/2009 - 07:05 by Shruti Sharma

Flanders, Belgium -- Flemish scientists say they have developed an improved and safer technique to deliver genes into the body's cells during genetic therapy.

Gene therapy is the introduction of genetic material into a patient's cells resulting in a cure or a therapeutic effect, said researchers at the Flanders Institute for Biotechnology in Belgium. The success of gene therapy ultimately depends on the gene delivery vehicles, or vectors, and most vectors have been derived from viruses that can be tailor-made to deliver therapeutic genes. The drawback is some of the viral vectors can induce side effects, including cancer and inflammation.