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Contact: Jennifer Gatti, 914-740-2100,
Promising Safety and Efficacy Results With Novel Lentiviral-Based Gene Therapy for SCID
New Rochelle, NY, December 1, 2016—A novel gene therapy-based approach for treating severe combined immunodeficiency disorder (SCID) involves transplanting a patient's own hematopoietic stem cells that have been transduced with a lentiviral vector carrying a correct copy of the mutated, SCID-causing gene. The promising safety and efficacy results achieved with this gene addition strategy support the initiation of a clinical trial in children with ART-SCID and are reported 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 December 31, 2016.
The article entitled "Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency," describes the design and use of a lentiviral gene delivery vector that expresses the Artemis gene, which is mutated in Artemis-SCID.
Divya Punwani and coauthors from University of California School of Medicine and University of California San Francisco Children's Hospital, University of California Los Angeles, University of Minnesota, Indiana University (Indianapolis), Hannover Medical School (Germany), and the National Institute of Allergy and Infectious Diseases (NIH, Bethesda, MD) showed that autologous hematopoietic cell transplantation (HCT) can overcome many of the problems associated with the use of donor stem cells in patients with Artemis-SCID.
“Lentivirus-based gene therapy has already had a transformative impact on treatment of patients with a number of genetic disorders of hematopoietic stem and progenitor cells. This report demonstrates that Artemis-SCID patients may also benefit from this approach,” 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 Institute of Allergy and Infectious Diseases, Division of Intramural Research and Warren Magnusen Clinical Center. 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, 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.