Biology News Net - Gene Therapy

Gene therapy in a droplet could treat eye diseases, prevent blindness

Wed, 27 Jul 2016 17:24:15 -0500

Eye diseases such as diabetic retinopathy and age-related macular degeneration are among the leading causes of irreversible vision loss and blindness worldwide. Currently, gene therapy can be administered to treat these conditions -- but this requires an injection. Now researchers report in the journal ACS Applied Materials & Interfaces a new way to deliver the treatment topically, without a needle.

Scientist develops gene therapy for muscle wasting

Wed, 27 Jul 2016 17:24:15 -0500

A discovery by Washington State University scientist Dan Rodgers and collaborator Paul Gregorevic could save millions of people suffering from muscle wasting disease.

New targeted gene therapy could lead to improved treatment for emphysema

Tue, 05 Jul 2016 17:36:32 -0500

Researchers have developed a new strategy using lung-targeted gene therapy that may lead to improved treatments for inherited diseases including emphysema.

Gene therapy shows long-term benefit for treating rare blindness

Thu, 28 Apr 2016 16:59:27 -0500

Pioneering gene therapy has restored some vision to patients with a rare form of genetic blindness for as long as four years, raising hopes it could be used to cure common causes of vision loss, new University of Oxford research published today shows.

Gene therapy: A promising candidate for cystic fibrosis treatment

Mon, 16 Nov 2015 21:05:56 -0500

An improved gene therapy treatment can cure mice with cystic fibrosis (CF). Cell cultures from CF patients, too, respond well to the treatment. Those are the encouraging results of a study presented by the Laboratory for Molecular Virology and Gene Therapy at KU Leuven, Belgium.

Gene therapy treats all muscles in the body in muscular dystrophy dogs

Thu, 22 Oct 2015 15:41:44 -0500

Muscular dystrophy, which affects approximately 250,000 people in the U.S., occurs when damaged muscle tissue is replaced with fibrous, fatty or bony tissue and loses function. For years, scientists have searched for a way to successfully treat the most common form of the disease, Duchenne Muscular Dystrophy (DMD), which primarily affects boys. Now, a team of University of Missouri researchers have successfully treated dogs with DMD and say that human clinical trials are being planned in the next few years.

Gene therapy fully restores vision in mouse model of Leber congenital amaurosis

Mon, 31 Aug 2015 14:34:43 -0500

Mice lacking the protein retGC1, which is deficient in humans suffering Leber congenital amaurosis-1 (LCA1), a disorder that causes severe visual impairment beginning in infancy, received gene therapy to replace retGC1 and showed fully restored visual function that persisted for at least 6 months. The success of this approach strongly support clinical testing of a gene therapy targeted to the retinas of LCA1 patients, conclude the authors of the study published in Human Gene Therapy. The article is available free on the Human Gene Therapy website until September 30, 2015.

Degenerating neurons respond to gene therapy treatment for Alzheimer's disease

Thu, 27 Aug 2015 16:31:21 -0500

Degenerating neurons in patients with Alzheimer's disease (AD) measurably responded to an experimental gene therapy in which nerve growth factor (NGF) was injected into their brains, report researchers at University of California, San Diego School of Medicine in the current issue of JAMA Neurology.

New gene therapy method provides specific, safe control of therapeutic transgenes

Mon, 13 Jul 2015 19:06:26 -0500

Korean researchers have described a novel control system to regulate the expression of a therapeutic transgene by targeting the passenger strand of a microRNA (miR-122) linked to the transgene. The researchers report that a control system based on targeting the passenger strand of miR-122 rather than the guide strand can regulate expression of an exogenous, therapeutic gene, while not affecting the expression of endogenous genes, in an article in Nucleic Acid Therapeutics. The article is available free on the Nucleic Acid Therapeutics website until August 13, 2015.

Gene therapy clips out heart failure causing gene mutations

Wed, 29 Apr 2015 19:13:25 -0500

This image shows the heart failure gene editing process. Gene therapy can clip out genetic material linked to heart failure and replace it with the normal gene in human cardiac cells, according to a study led by researchers from the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai. The study is published in the April 29 edition of Nature Communications.