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Encouraging Gene Therapy Results in Genetic Form of Blindness

NEW ROCHELLE, N.Y., Sept. 8 /PRNewswire/ -- All three patients who received intraocular adeno-associated virus-based gene therapy to treat Leber congenital amaurosis (LCA), an incurable genetic disorder that results in blindness, had improved vision, according to a paper published online ahead of print in Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc. These promising results provided evidence of therapeutic efficacy, complementing two other recent LCA clinical trial reports in the New England Journal of Medicine. The Human Gene Therapy paper is available free online at

LCA can be caused by many different genes but the newly treated form is caused by mutations in the RPE65 gene, which is essential for maintenance of sight. A group of researchers from the University of Pennsylvania (Philadelphia) and University of Florida (Gainesville) injected copies of a normal RPE65 gene into one eye each of three young adults with LCA. The patients underwent follow-up examinations for 90 days to assess changes in the eye and in their vision compared to the untreated eye. The replacement RPE65 gene was packaged in a gene therapy vector engineered from a recombinant adeno-associated virus.

All of the patients self-reported improved vision under dim light conditions, and the authors measured significant increases in dark-adapted visual sensitivity in the treated eye after therapy compared to before therapy; the untreated eyes did not change.

In a paper entitled, "Phase I Trial of Leber Congenital Amaurosis due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results," the authors reported no vector-related serious adverse events, immune reactions, or systemic toxicity.

"Human ocular gene therapy for inherited retinal disease is undergoing a birth experience as early phase trials report efficacy and safety. LCA can now be redefined as a potentially curable eye disease," said Samuel G. Jacobson MD, PhD, Professor of Ophthalmology at the University of Pennsylvania and Principal Investigator of the study. "This is a major source of encouragement to the patients and to all the scientists and clinicians who have spent decades building the knowledge base to the level where we could begin these clinical trials."

Human Gene Therapy, the Official Journal of the European Society of Gene and Cell Therapy (, the British Society for Gene Therapy (, and the French Society of Cell and Gene Therapy ( is an authoritative peer-reviewed journal published monthly in print and online that presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Tables of contents and a free sample issue may be viewed online at

Mary Ann Liebert, Inc (, is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Tissue Engineering, Stem Cells and Development, and Cloning and Stem Cells. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 60 journals, books, and newsmagazines is available at

Mary Ann Liebert, Inc. 140 Huguenot St., New Rochelle, NY 10801-5215

Phone: (914) 740-2100 (800) M-LIEBERT Fax: (914) 740-2101

Contact: Vicki Cohn, Mary Ann Liebert, Inc, (914) 740-2100, ext. 2156,

SOURCE Mary Ann Liebert, Inc.
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