Gene therapy for lysosomal storage disease shown to be safe and well tolerated

(Press-News.org) New Rochelle, NY, March 11, 2014—Several young children suffering from a severe degenerative genetic disease received injections of therapeutic genes packaged within a noninfectious viral delivery vector. Safety, tolerability, and efficacy results from this early stage clinical trial are reported in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available on the Human Gene Therapy website.

Marc Tardieu, Université Paris-Sud and INSERM, and a team of international researchers administered the adeno-associated viral (AAV) vector carrying a normal copy of the N-sulfoglycosamine sulfohydrolase (SGSH) gene into the brains of four children affected by mucopolysaccharidosis type IIIA (MPSIIIA), an inherited lysosomal storage disease in which the SGSH gene is defective. The AAV vector also delivered a sulfatase-modifying factor (SUMF1), needed to activate the SGSH protein.

In addition to measures of toxicity, adverse events, and tolerability, the researchers evaluated the children for brain shrinkage (a characteristic of MPSIIIA) and for changes in behavior, attention, sleep, and cognitive benefit. They describe their findings in the article "Intracerebral administration of AAV rh.10 carrying human SGSH and SUMF1 cDNAs in children with MPSIIIA disease: results of a phase I/II trial." "This is an important new approach for treating CNS manifestations of lysosomal storage diseases that could be applied across a wide array of disorders," says James M. Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.

INFORMATION: 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. 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 sister journals, Human Gene Therapy Methods, published bimonthly and 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 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.

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