Public Release: 

Long-term efficacy of AAV5-based gene therapy to treat day blind sheep with achromatopsia

Mary Ann Liebert, Inc./Genetic Engineering News

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IMAGE: Human Gene Therapy presents reports on the transfer and expression of genes in mammals, including humans. view more 

Credit: Mary Ann Liebert, Inc., publishers

New Rochelle, NY, August 15, 2018--A study of a large animal model of achromatopsia caused by a mutation in the CNGA3 gene that was treated with a single injection of CNGA3 gene therapy delivered using an AAV5 vector revealed findings reported long-term follow-up findings that show promise for the efficacy and safety of this therapeutic approach. The results demonstrated significant improvement in cone function and no abnormalities in the treated eyes for up to six years following delivery of the CNGA3 transgene, as reported in an article published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. Click here to read the full-text article free on the Human Gene Therapy website through September 15, 2018.

"Six Years and Counting: Restoration of Photopic Retinal Function and Visual Behavior Following Gene Augmentation Therapy in a Sheep Model of CNGA3 Achromatopsia" describes the recovery of cone photoreceptor function, as determined by photopic behav-ioral maze tests and electroretinographic (ERG) examinations. The treated sheep had pas-sage times on the photopic maze test and numbers of collisions that were significantly lower than pre-treatment values at all follow-up exams. ERG testing showed significant improvement throughout the follow-up period.

Ron Ofri, Koret School of Veterinary Medicine, The Hebrew University of Jerusalem (Rehovot, Israel) and Edward Averbukh, Hadassah-Hebrew University Medical Center (Jerusalem, Israel) led a team of researchers from The Hebrew University of Jerusalem, Hadassah-Hebrew University Medical Center, Agricultural Research Organization (Beit Dagan, Israel), and University of Florida (Gainesville) as coauthors of the article.

"Confirmation of the concept of 'once-in-a-lifetime' gene therapy for genetic blindness depends on patient, careful studies such as these," says Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, Worces-ter, MA. "Human gene therapy investigators in the field of retinal diseases should find these results very encouraging, as they move forward in expanding clinical applications of the platform of rAAV gene therapy."

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About the Journal

Human Gene Therapy, the Official Journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies, is an authoritative peer-reviewed journal published monthly in print and online. Led by Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, Human Gene Therapy 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. Its companion journals, Human Gene Therapy Methods, published bimonthly, focuses on the application of gene therapy to product testing and development, and Human Gene Therapy Clinical Development, published quarterly, features data relevant to the regulatory review and commercial development of cell and gene therapy products. Tables of contents for all three publications and a free sample issue may be viewed on the Human Gene Therapy website.

About the Publisher

Mary Ann Liebert, Inc., publishers 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 Nucleic Acid Therapeutics, Tissue Engineering, Stem Cells and Development, and Cellular Reprogramming. Its biotechnology trade magazine, GEN (Genetic Engineering & Biotechnology News), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 80 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.

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