Gene therapy prevents disorders with alcohol exposure in ALDH2 deficiency



Credit: Mary Ann Liebert, Inc., publishers

New Rochelle, NY, February 12, 2020–A new study has shown that gene therapy to treat one of the most common hereditary disorders, aldehyde dehydrogenase type 2 (ALDH2) deficiency, may prevent increased risk for esophageal cancer and osteoporosis associated with chronic alcohol exposure. The study, performed in a mouse model of ALDH2, is 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 March 12, 2020.

Ronald Crystal, Matthew Greenblatt, Katie Stiles, and colleagues from Weill Cornell Medical College, The Rockefeller University, and Hospital for Special Surgery, New York, NY, coauthored the article entitled “Systemic Adeno-Associated Virus-Mediated Gene Therapy Prevents the Multiorgan Disorders Associated with Aldehyde Dehydrogenase 2 Deficiency and Chronic Ethanol Ingestion.” The researchers delivered the ALDH2 gene to two mouse models of ALDH2 deficiency using an adeno-associated virus (AAV) vector. Some of the mice were then given ethanol in their drinking water over a 12-week period.

In ALDH2 deficiency, proper ethanol metabolism is not possible, and the systemic accumulation of acetaldehyde causes acute abnormalities as well as chronic disorders including esophageal damage that can lead to the development of esophageal cancer, and abnormalities in bone metabolism that can lead to osteoporosis. Compared to the non-ethanol drinking mice, the mice treated with gene therapy who ingested alcohol did not show signs of either the acute abnormalities or the chronic disorders normally associated with ethanol exposure in ALDH2 deficiency.

“This work by Dr. Crystal and his collaborators points to a new potential therapy for individuals with a particular genetic susceptibility to suffer long-term consequences of excessive drinking,” 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 under Award Numbers R41 AA027730 01 and DP5OD021351. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

About the Journal

Human Gene Therapy, the Official Journal of the European Society of Gene and Cell Therapy and eight other international gene therapy societies, was the first peer-reviewed journal in the field and provides all-inclusive access to the critical pillars of Human Gene Therapy: research, methods, and clinical applications. The Journal is 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, and an esteemed international editorial board. Human Gene Therapy is available in print and online. Complete tables of contents and a sample issue are available 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.

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