Major advances and ongoing challenges for gene therapy in SCID-X1


New Rochelle, NY, February 29, 2016–X-linked severe combined immunodeficiency (SCID-X1) was the test-case for demonstrating the feasibility of using gene therapy targeting hematopoietic stem cells to reconstitute the human immune system and provide effective long-term treatment for a hereditary disease. A comprehensive and forward-looking review of the biological and medical research that helped advance this field is captured in the article "Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?", published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free to read on the Human Gene Therapy website until March 31, 2016.

The article is part of a special issue of Human Gene Therapy focusing on advances in gene and cell therapy research in France, led by Guest Editors Nathalie Cartier, MD, Director of Research, INSERM, Paris, and President, European Society of Gene and Cell Therapy (ESGCT), and Pierre Cordelier, PhD, Senior Researcher, INSERM, Toulouse, France, and President, French Society of Cell and Gene Therapy (SFTCG). The special issue will be distributed at the SFTCG meeting, March 9-11, Marseilles, France.

Marina Cavazzana, Emmanuelle Six, Chantal Lagresle-Peyrou, Isabelle André-Schmutz, and Salima Hacein-Bey-Abina, Assistance Publique-Hopitaux de Paris, INSERM, Imagine Institute, Université Paris Descartes, and Groupe Hospitalier Universitaire Paris-Sud, Paris, France, put into perspective the clinical advances in gene therapy for SCID-X1, tracing the history of clinical trials and describing the development and implementation of new and safer gene delivery vectors. The researchers also describe how increasing knowledge of the integrome has helped advance the field, examine remaining challenges such as improved safety and long-term immune reconstitution, and explore how progress in treating SCID-X1 has contributed to applications of gene therapy in other hereditary diseases.

"The French have been the true pioneers of gene therapy for SCID-X1, proving that hematopoietic stems cells are a suitable platform for cure of genetic diseases," 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.


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.

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