Promising results of gene therapy to treat diseases of the eye
The easy accessibility of the eye and the established link between specific genetic defects and ocular disorders offer hope for using gene therapy to provide long-term therapeutic benefit. Two reports in the current issue of Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc. (www.liebertpub.com), describe the effective replacement of a human gene to preserve photoreceptor function in a mouse model of severe retinal degeneration. The articles are available free online (www.liebertpub.com/hum). Basil Pawlyk and colleagues from Harvard Medical School and Massachusetts Eye and Ear Infirmary (Boston, MA) delivered the human gene for RGPR-interacting protein-1 to mice affected with Leber congenital amaurosis (LCA), a condition linked to a mutated form of RPGRIP1 that causes degeneration of photoreceptors in the eye. The researchers packaged the gene in an adeno-associated virus (AAV) vector and injected the vector under the retinas of the affected mice. They demonstrated expression of the human gene in the photoreceptors, with correct localization to the cilia. Further evaluation revealed improved function and survival of the photoreceptors in the treated eyes.
The authors conclude that the results of this study, presented in the paper entitled, "Replacement Gene Therapy with a Human RPGRIP1 Sequence Slows Photoreceptor Degeneration in Murine Model of Leber Congenital Amaurosis," validate a gene therapy design that could serve as the basis for a future clinical trial in patients affected by this form of LCA.
In the same issue, Kamolika Roy, Linda Stein, and Shalesh Kaushal from University of Massachusetts Medical School (Worcester) review the use of recombinant AAV vectors for gene therapy to treat ocular diseases. Based on the success of three early-stage clinical trials in LCA, they conclude that this approach appears "to be a safe, effective, and long-term treatment for LCA, a previously untreatable disorder." In the article, "Ocular Gene Therapy: An Evaluation of Recombinant Adeno-Associated Virus-Mediated Gene Therapy Interventions for the Treatment of Ocular Disease," they conclude that rAAV-mediated gene therapy is "the most suitable gene therapy treatment approach for ocular diseases."
"The successful correction of this photoreceptor defect in a relevant mouse model of LCA should usher in a new wave of translational research in retinal degeneration syndromes," says James M. Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy, and Head of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine, in Philadelphia.
Articles on the same topic
- Breakthrough gene therapy prevents retinal degenerationMon, 16 Aug 2010, 10:15:52 EDT
- Breakthrough gene therapy prevents retinal degenerationfrom Biology News NetTue, 17 Aug 2010, 1:35:10 EDT
- Breakthrough gene therapy prevents retinal degenerationfrom Science DailyMon, 16 Aug 2010, 13:35:17 EDT
- Breakthrough gene therapy prevents retinal degenerationfrom PhysorgMon, 16 Aug 2010, 11:42:11 EDT
- Promising results of gene therapy to treat diseases of the eyefrom Science DailySun, 15 Aug 2010, 23:14:19 EDT
- Promising results of gene therapy to treat diseases of the eyefrom PhysorgSat, 14 Aug 2010, 3:35:10 EDT
- Promising results of gene therapy to treat diseases of the eyefrom Science BlogFri, 13 Aug 2010, 18:35:11 EDT
Latest Science NewsletterGet the latest and most popular science news articles of the week in your Inbox! It's free!
Learn more about
Check out our next project, Biology.Net
From other science news sites
Popular science news articles
- Russian physicists discover a new approach for building quantum computers
- Eastern Pacific storms Georgette and Frank see-saw in strength
- SwRI-led study shows puzzling paucity of large craters on dwarf planet Ceres
- Task force maybe too stringent in not yet recommending melanoma screening
- Evidence insufficient to make recommendation regarding visual skin examination by a clinician
- Three-drug combinations could help counter antibiotic resistance, UCLA biologists report
- Patch delivers drug, gene, and light-based therapy to tumor sites
- Scientists develop painless and inexpensive microneedle system to monitor drugs
- Unusual new zoantharian species is the first described solitary species in over 100 years
- Living on borrowed time
- Researchers create means to monitor anthropogenic global warming in real time
- Hundreds of years later, teeth tell the story of people who didn't get enough sunshine
- House-hunting ants know how to take the hassle out of moving
- Research team led by NUS scientists develop plastic flexible magnetic memory device
- Ship engine emissions adversely affect macrophages