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Progress and promise of gene transfer and gene editing to cure beta-thalassemias

May 09, 2016

Promising results from the first clinical trials of globin gene transfer to treat beta-thalassemias-inherited forms of anemia-have eliminated the need for blood transfusions in some individuals. Enhancing current gene therapy strategies and applying new gene editing tools to correct beta-globin deficiencies and to reactivate fetal hemoglobin production are among the exciting new advances being pursued in the search for a cure for severe globin disorders, as described 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 June 5, 2016.

Jorge Mansilla-Soto, Isabelle Riviere, Farid Boulad, and Michel Sadelain, Memorial Sloan Kettering Cancer Center, New York, NY, are coauthors of the article "Cell and Gene Therapy for the Beta-Thalassemias: Advances and Prospects." They present the rationale for using globin gene therapy to cure beta-thalassemias and discuss the four Phase I clinical studies underway in the U.S. and Europe. The authors also highlight the important role that emerging gene editing and gene repair technologies can have in correcting beta-globin deficiencies. The possibility for a genetic intervention to lead to a cure for the beta-thalassemias is increasingly likely the researchers conclude.

This article is part of a Festschrift in honor of George Stamatoyannopoulous, MD, DrSci, Professor of Medicine and Genome Sciences, and Director, Markey Molecular Medicine Center, University of Washington, Seattle.

"The review from the Sadelain lab highlights the tremendous progress in gene therapy for hemoglobin disorders," 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. "It is very fitting that it was authored by such a prominent translational physician-scientist who was inspired by Dr. Stam."

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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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