10:18am Tuesday 21 November 2017

Alternative Strategy for Gene Replacement Shows Promise in Duchenne Muscular Dystrophy

The article is available free on the Human Gene Therapy website until October 29, 2015.

Kristin Heller, Louise Rodino-Klapac, and coauthors from The Research Institute at Nationwide Children’s Hospital and The Ohio State University, Columbus, describe the method they used to deliver the α7 gene, which codes for the ITGA7 laminin receptor, to 5–7 day old mice deficient in both dystrophin and utrophin. These mice would usually die between 6–20 weeks of age, but gene transfer of α7 extended longevity by more than 10 weeks, as described in the article “Human α7 Integrin Gene (ITGA7) Delivered by Adeno-Associated Virus Extends Survival of Severely Affected Dystrophin/Utrophin-Deficient Mice.”

“Recombinant AAV vectors are showing signs of clinical efficacy in a number of genetic disorders,” 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. “Certain genetic and immunologic aspects of Duchenne muscular dystrophy have made it a very complex target for simple rAAV-mediated gene replacement. This alternative strategy holds great promise for circumventing those potential issues to achieve clinical benefit in these patients.”  

Click here to share this release with your colleagues.

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, Worcester, MA. 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 content 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, 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 80 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.


Share on:
or:

MORE FROM Genetics and Birth Defects

Health news