AAV9 gene therapy vector dramatically increases life span in krabbe disease mouse model

August 14, 2019

New Rochelle, NY, August 12, 2019--An optimized and newly engineered form of the adeno-associated vector 9 (AAV9) vector used to deliver the galactosylceramidase gene to a mouse model of the inherited neurogenerative and rapidly fatal form of Krabbe dis-ease improved clinical symptoms and prolonged median survival by 275%. Two-day old mice treated with a single injection of the systemic gene therapy had a significant increase in median life span to 150 days, compared to 41 days for the untreated mice, as reported in the study 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 12, 2019.

The article entitled "An Engineered Galactosylceramidase Construct Improves AAV Gene Therapy for Krabbe Disease in Twitcher Mice" was coauthored by Dongsheng Duan, Steven LeVine, and colleagues from the University of Missouri (Columbia), Uni-versity of Missouri School of Medicine and College of Veterinary Medicine, and Univer-sity of Kansas Medical Center (Kansas City).

The researchers describe the construction and characterization of the specially engineered AAV9 vector, built on the codon-optimized mouse galactosylceramidase coding se-quence, and designed for improved protein delivery to the central nervous system and enhanced secretion of the galactosylceramidase enzyme. At 5 weeks of age, the treated mice had better body weight and motor function than the untreated mice. The longest-lived treated mouse survived to 180 days.

"This work shows dramatic in vivo proof-of-principle, indicating a robust therapeutic ef-fect from this optimized vector," 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 un-der Award NumbersP30HD02528, 9P2OGM104936, and S10RR027564. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.
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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., publisher website.

Mary Ann Liebert, Inc./Genetic Engineering News

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