New gene therapy strategies emerging to combat vision loss
New Rochelle, NY, June 22, 2016–Diseases of the eye that cause vision loss and blindness, especially neurodegenerative disorders affecting the retina, are ideal targets for gene therapy, including gene replacement and promising corrective gene editing strategies. A comprehensive Review article providing an overview of emerging therapeutic approaches and innovative gene delivery and gene editing tools to treat ocular diseases is published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free for download on the Human Gene Therapy website.
Lolita Petit, Hemant Khanna, and Claudio Punzo, University of Massachusetts Medical School, Worcester, coauthored the article "Advances in Gene Therapy for Diseases of the Eye." They provide insights on recent progress in clinical applications of retinal gene replacement therapy, preclinical advances in gene-specific therapy for photoreceptor diseases, and the development of gene independent therapeutic strategies.
"The spectacular successes of gene therapy for eye diseases may be seen as providing a platform for molecular approaches to a much broader range of disorders affecting vision," 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, Worcester, MA.
Research reported in this publication was supported by the National Institutes of Health under Award Numbers EY022372 and EY023570. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.
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.