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Promising results reported in study of AAV-based gene delivery to treat methylmalonic acidemia

April 06, 2016

A new study in patients with the inherited metabolic disorder methylmalonic academia (MMA) found lower than expected levels of antibodies against the adeno-associated viral (AAV) vectors being developed for gene therapy to replace the enzyme (MUT) that is deficient in MMA. These results, and especially the absence of AAV antibodies in most patients who completely lack the MUT enzyme and have not had a liver transplant--those in greatest need of improved therapies--has encouraging implications for gene delivery as a treatment for MMA, as described in an article 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 until May 1, 2016.

Elizabeth Harrington, Jennifer Sloan, Irini Manoli, Randy Chandler, Charles Venditti, and Peter McGuire, National Human Genome Research Institute, National Institutes of Health (Bethesda, MD), and Mark Schneider, Roberto Calcedo, and James Wilson, Perelman School of Medicine, University of Pennsylvania (Philadelphia), coauthored the article entitled "Neutralizing Antibodies Against Adeno-Associated Viral Capsids in Patients with mut Methylmalonic Acidemia (MMA)".

AAV is proving to be an effective gene transfer vehicle for systemic gene delivery. The authors have developed and tested AAV vectors that carry the mouse mut gene and have reported promising results of gene transfer in a mouse model of MMA. They have also developed an AAV vector to deliver the human MUT gene. The presence of pre-existing antibodies to AAV capsid proteins can trigger an immune reaction against the vector and interfere with delivery of the therapeutic gene.

"The presence of neutralizing antibodies in some patients is one of the few remaining obstacles to successful correction of genetic diseases with AAV vectors," 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. "The observation that anti-AAV antibody levels are lower than expected in MMA patients bodes well for the future success of AAV-based gene therapy with this disorder."

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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.

Mary Ann Liebert, Inc./Genetic Engineering News


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