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
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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All abstracts accepted for presentation at the ARVO Annual Meeting represent previously unpublished data and conclusions.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.

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Association for Research in Vision and Ophthalmology

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