New work from Ron Crystal’s lab on treating hereditary adrenal disorders

New Rochelle, NY, March 29, 2018–A new study has definitively shown that a single treatment with gene therapy using adeno-associated viral (AAV) vector gene delivery to replace the defective gene responsible for congenital adrenal hyperplasia (CAH) will only temporarily alleviate the hereditary disorder. The normal cells will eventually be replaced by new adrenocortical cells carrying the disease mutation, and a long-term solution will require targeting the genetic mutation carried in a patient's adrenocortical stem cells, as reported in an article published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Human Gene Therapy website until April 29, 2018.

Sandra Markmann, Ronald Crystal, and a team of researchers from Weill Cornell Medical College, New York, NY coauthored the article entitled "Biology of the Adrenal Gland Coretex Obviates Effective Use of Adeno-Associated Virus Vectors to Treat Hereditary Adrenal Disorders ." The researchers demonstrated that the loss over time of therapeutic genes delivered by AAV vectors was not due to an immune response against the AAV delivery vehicles and was instead a result of the renewal of adrenocortical stem cell populations.

"There has been a growing trend in gene therapy to target diseases identified by newborn screening. CAH screening has been included in the U.S. for decades, with corticosteroid replacement being the only therapy," 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. "Defining the limitations to AAV gene therapy in such disorders is an important step, much as was the early gene therapy work in cystic fibrosis. Such studies, even though they show limitations, move us closer to identifying the right solution for these patients."

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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. 140 Huguenot St., New Rochelle, NY 10801-5215 http://www.liebertpub.com Phone: 914-740-2100 (800) M-LIEBERT Fax: 914-740-2101

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Original Source

https://home.liebertpub.com/news/new-work-from-ron-crystals-lab-on-treating-hereditary-adrenal-disorders/2359 http://dx.doi.org/10.1089/hum.2017.203