Navigation Links
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.
Copyright©2008 PR Newswire.
All rights reserved

Related medicine technology :

1. Multiple NIH Phase 2 HIV Vaccine Trials Using Vicals Technology Produce Encouraging Results
2. Pharmions Amrubicin Shows Encouraging Results Compared to Standard of Care in Second Line Treatment of Small Cell Lung Cancer
3. Actress and Mother Jennifer Garner Kicks Off American Lung Associations Faces of Influenza Awareness Campaign, Encouraging Families to Get Vaccinated Against Influenza
4. New UNAIDS Report Offers Encouraging News, But Most HIV-Positive Mothers Still Not Getting the Care & Treatment They Need
5. Exelixis Presents Encouraging Phase 1 Data For XL019, A Novel Selective Inhibitor of JAK2
6. PTC Therapeutics Presents Encouraging Phase 1 Results of its Novel VEGF Inhibitor, PTC299, at 30th Annual San Antonio Breast Cancer Symposium
7. Aeras, Crucell and South African Tuberculosis Vaccine Initiative Announce Encouraging Preliminary Results of Tuberculosis Vaccine Clinical Trial in South Africa
8. Exelixis Reports Encouraging Phase 1 Data for XL184 at ASCO
9. EDAP Announces Launch of Clinical Study Combining HIFU and Chemotherapy for Localized Aggressive High Risk Prostate Cancer
10. Indevus Receives Approvable Letter From FDA for VALSTAR(R) for Bladder Cancer Therapy
11. Progesterone Therapy and Preterm Birth: More Evidence Helps Identify Women Who Can Benefit
Post Your Comments:
(Date:6/23/2016)... Roche (SIX: RO, ROG; OTCQX: RHHBY) announced that it ... (procalcitonin) assay as a dedicated testing solution for people ... Roche is the first IVD company in the U.S ... assessment and management. PCT is a sepsis-specific ... blood can aid clinicians in assessing the risk of ...
(Date:6/23/2016)... 2016 Bracket , a leading clinical trial ... clinical outcomes platform, Bracket eCOA (SM) 6.0, at the ... – 30, 2016 in Philadelphia , Pennsylvania.  ... Assessment product of its kind to fully integrate with RTSM, ... eCOA 6.0 is a flexible platform for electronic clinical outcomes ...
(Date:6/23/2016)... Revolutionary technology includes multi-speaker listening to conquer ... in advanced audiology and hearing aid technology, has today ... world,s first internet connected hearing aid that opens up ...      (Photo: ) , ... , TwinLink™ - the first dual communication ...
Breaking Medicine Technology:
(Date:6/26/2016)... , ... June 27, 2016 , ... Quality metrics are ... in many ways they remain in the eye of the beholder, according to experts ... publication of The American Journal of Managed Care. For the full issue, click ...
(Date:6/26/2016)... Carolina (PRWEB) , ... June 26, 2016 , ... ... of a new product that was developed to enhance the health of felines. The ... centuries. , The two main herbs in the PawPaws Cat Kidney Support ...
(Date:6/25/2016)... ... 25, 2016 , ... Experts from the American Institutes for ... Meeting June 26-28, 2016, at the Hynes Convention Center in Boston. , AIR ... care planning, healthcare costs and patient and family engagement. , AIR researchers will ...
(Date:6/25/2016)... ... ... "With 30 hand-drawn hand gesture animations, FCPX users can easily customize each ... Film Studios. , ProHand Cartoon’s package transforms over 1,300 hand-drawn pictures into hand ... select a ProHand generator and drag it above media or text in the Final ...
(Date:6/25/2016)... ... June 25, 2016 , ... Dr. Calvin ... injury. Recently, he has implemented orthobiologic procedures as a method for treating his ... of the first doctors to perform the treatment. Orthobiologics are substances that orthopaedic ...
Breaking Medicine News(10 mins):