Navigation Links
Gene therapy sets stage for new treatments for inherited blindness, Penn veterinary researchers say

PHILADELPHIA - Veterinary vision scientists at the University of Pennsylvania have safely and successfully used a viral vector in targeting a class of photoreceptors of the retina called rods, a critical first step in developing gene therapies for inherited blindness caused by rod degeneration.

In this study, the viral vector, or missile that carries the genetic material designed to correct a DNA mutation, was not intended to treat a disease but to demonstrate through the use of a fluorescent protein that a safe and effective viral cocktail could be delivered inside rod cells.

The next major challenge that vision researchers face is to target these photoreceptor cells for treatment, as the majority of retinal degenerative diseases are caused by mutations that damage these cells. Photoreceptors are the cells in the retina responsible for capturing light and transforming it into an electrical signal that will be interpreted by the brain.

A consortium of researchers from Penn and the University of Florida used a specific adeno-associated virus, AAV2/5, to carry the gene of a green fluorescent protein. The scientists tested whether three different promoters, pieces of DNA that play the role of a switch, could turn on the production of the green fluorescent protein in rods in dogs. Two of the three viral cocktails were able to turn on the production of the green fluorescent protein specifically in rods, while the third viral cocktail targeted rods, cones and other retinal cells; however only the proper concentration of each viral vector proved to be just right. Efficient and specific rod transduction, together with preservation of retinal structure, was achieved with both mOP and hGRK1 promoters when viral titers in the order of 1011 vg/ml were used.

"Now that we've demonstrated this type of delivery can be accomplished, " said William A. Beltran, assistant professor of ophthalmology at Penn's School of Veterinary Medicine and director of the study, "the next step is to target common rod degenerations using canine models that mimic the human diseases. The delivery of a viral vector, coupled with a rod-specific promoter is likely to be the safest and most efficient approach to correct diseases such as X-linked and autosomal dominant retinitis pigmentosa, both of which lead to complete blindness."

A form of childhood blindness called RPE65-Leber's congenital amaurosis, LCA, is the first and to this date the only inherited retinal disease for which corrective gene therapy is currently being tested in three on-going clinical trials. This treatment was first developed and perfected in dogs by some of the scientists who participated in the present study before its use in human patients.

The study appears in the current issue of the Journal of Gene Therapy.


Contact: Jordan Reese
University of Pennsylvania

Related biology news :

1. Smoking during radiation therapy for head and neck cancers linked to poorer outcomes
2. JDRF partners with LCT to test encapsulated cell replacement therapy for the treatment of diabetes
3. Concord Medical Enters Agreement to Acquire Four Radiotherapy and Diagnostic Imaging Centers in Hebei Province
4. Lengthening time a drug remains bound to a target may lead to improving diagnostics, therapy
5. Gene therapy cures canines of inherited form of day blindness, Penn veterinary researchers say
6. Exercise therapy for low back pain
7. Making the blind see: Gene therapy restores vision in mice
8. New cancer therapy may fight cardiovascular disease
9. Cancer therapy using unique imaging, delivery system focus of NSF CAREER Award
10. Development of new anti-cancer gene therapy approach using lentiviral vectors
11. Hormone therapy for food poisoning bacteria
Post Your Comments:
Related Image:
Gene therapy sets stage for new treatments for inherited blindness, Penn veterinary researchers say
(Date:4/15/2016)...  A new partnership announced today will help ... in a fraction of the time it takes ... life insurance policies to consumers without requiring inconvenient ... Diagnostics, rapid testing (A1C, Cotinine and HIV) and ... weight, pulse, BMI, and activity data) available at ...
(Date:3/31/2016)... , March 31, 2016 ... ) ("LegacyXChange" or the "Company") LegacyXChange is ... users of its soon to be launched online site ... ) will also provide potential shareholders ... of DNA technology to an industry that is notorious ...
(Date:3/22/2016)... , March 22, 2016 ... report "Electronic Sensors Market for Consumer Industry by Type ... Others), Application (Communication & IT, Entertainment, Home ... Global Forecast to 2022", published by MarketsandMarkets, ... expected to reach USD 26.76 Billion by ...
Breaking Biology News(10 mins):
(Date:6/27/2016)... , June 27, 2016  Liquid Biotech ... announced the funding of a Sponsored Research Agreement ... circulating tumor cells (CTCs) from cancer patients.  The ... in CTC levels correlate with clinical outcomes in ... These data will then be employed to support ...
(Date:6/24/2016)... , ... June 24, 2016 , ... While the majority ... as the Cary 5000 and the 6000i models are higher end machines that use ... height of the spectrophotometer’s light beam from the bottom of the cuvette holder. ...
(Date:6/23/2016)... June 23, 2016   Boston Biomedical , ... compounds designed to target cancer stemness pathways, announced ... granted Orphan Drug Designation from the U.S. Food ... gastric cancer, including gastroesophageal junction (GEJ) cancer. Napabucasin ... to inhibit cancer stemness pathways by targeting STAT3, ...
(Date:6/23/2016)... ... June 23, 2016 , ... Charm ... Mold) microbial test has received AOAC Research Institute approval 061601. , “This is ... last year,” stated Bob Salter, Vice President of Regulatory and Industrial Affairs. “The ...
Breaking Biology Technology: