Gene therapy used to preserve sight in patients

May 04, 2014

Orlando, Fla. --In two separate studies, vision scientists have developed healthy genes to prevent blinding diseases that stem from genetic defects. The research is being presented at the 2014 Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) this week in Orlando, Fla.

In a clinical trial to treat choroideremia, a rare disease that causes progressive and irreversible blindness, scientists developed a virus that can replace the missing gene (that causes the disease) in the cells at the back of the eye. Six months after the virus was injected into patients, findings showed that some patients experienced improved vision.

Abstract Title: Improved visual function in patients with choroideremia undergoing subretinal gene therapy

Presentation Start/End Time: Sunday, May 4, 3:15 - 3:30pm
Location: S 320AB
Session Number: 147

In a separate study, researchers developed a gene therapy to stop the progression of a form of retinitis pigmentosa, an inherited disease transmitted from mothers to sons. Two years after the therapy was used to treat dogs at an early stage of the disease, the treatment remained effective. Further use of the technique in dogs with mid and late stages of the disease also resulted in a positive response to the intervention.

Abstract Title: RPGR gene augmentation delivered at early, mid and late stage disease in a canine model of XLRP rescues photoreceptor structure and function

Presentation Start/End Time: Tuesday, May 6, 11am - 12:45pm
Location: Exhibit/Poster Hall SA
Session Number: 342
The Association for Research in Vision and Ophthalmology (ARVO) is the largest eye and vision research organization in the world. Members include some 11,500 eye and vision researchers from over 70 countries. ARVO encourages and assists research, training, publication and knowledge-sharing in vision and ophthalmology.

All abstracts accepted for presentation at the ARVO Annual Meeting represent previously unpublished data and conclusions. This research may be proprietary or may have been submitted for journal publication.

Embargo policy: Journalists must seek approval from the presenter(s) before reporting data from paper or poster presentations. Press releases or stories on information presented at the ARVO Annual Meeting may not be released or published until the conclusion of the presentation.

Visit us at:


Association for Research in Vision and Ophthalmology

Related Gene Therapy Articles from Brightsurf:

Risk of AAV mobilization in gene therapy
New data highlight safety concerns for the replication of recombinant adeno-associated viral (rAAV) vectors commonly used in gene therapy.

Discovery challenges the foundations of gene therapy
An article published today in Science Translational Medicine by scientists from Children's Medical Research Institute has challenged one of the foundations of the gene therapy field and will help to improve strategies for treating serious genetic disorders of the liver.

Gene therapy: Novel targets come into view
Retinitis pigmentosa is the most prevalent form of congenital blindness.

Gene therapy targets inner retina to combat blindness
Batten disease is a group of fatal, inherited lysosomal storage disorders that predominantly affect children.

New Human Gene Therapy editorial: Concern following gene therapy adverse events
Response to the recent report of the deaths of two children receiving high doses of a gene therapy vector (AAV8) in a Phase I trial for X-linked myotubular myopathy (MTM).

Restoring vision by gene therapy
Latest scientific findings give hope for people with incurable retinal degeneration.

Gene therapy/gene editing combo could offer hope for some genetic disorders
A hybrid approach that combines elements of gene therapy with gene editing converted an experimental model of a rare genetic disease into a milder form, significantly enhancing survival, shows a multi-institutional study led by the University of Pennsylvania and Children's National Hospital in Washington, D.C.

New technology allows control of gene therapy doses
Scientists at Scripps Research in Jupiter have developed a special molecular switch that could be embedded into gene therapies to allow doctors to control dosing.

Gene therapy: Development of new DNA transporters
Scientists at the Institute of Pharmacy at Martin Luther University Halle-Wittenberg (MLU) have developed new delivery vehicles for future gene therapies.

Gene therapy promotes nerve regeneration
Researchers from the Netherlands Institute for Neuroscience and the Leiden University Medical Center have shown that treatment using gene therapy leads to a faster recovery after nerve damage.

Read More: Gene Therapy News and Gene Therapy Current Events is a participant in the Amazon Services LLC Associates Program, an affiliate advertising program designed to provide a means for sites to earn advertising fees by advertising and linking to