Update on Advances in Gene Therapy From National Center for Advancing Translational Sciences

NEW ROCHELLE, N.Y. - Feb. 24, 2016 - PRLog -- New initiatives by the National Center for Advancing Translational Sciences (NCATS) to use gene therapy approaches to treat rare diseases and especially promising aspects of gene transfer and gene editing technology, such as adeno-associated viral (AAV) vectors and CRISPR-Cas9 are highlighted in an editorial published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers (http://www.liebertpub.com/). The article is available free for download on the Human Gene Therapy (http://online.liebertpub.com/doi/full/10.1089/hum.2016.29018.pjb) website.

In the article “Gene Therapy: The View from NCATS (http://online.liebertpub.com/doi/full/10.1089/hum.2016.29018.pjb),” Philip Brooks, N. Nora Yang, and Christopher Austin, National Institutes of Health (NIH), Bethesda, MD, describe NCATS’ strategy of focusing on platform approaches that can be readily adapted to develop treatments for multiple diseases. The aim is to identify the most promising methods and support their translation from the laboratory to the clinic so patients can benefit from these novel therapeutic strategies.

Among the recent development in gene therapy that are particularly relevant to NCATS’ mission and priorities are major advances in the development of viral vectors to treat rare diseases, including AAV, which offers significant advantages over adenovirus and retrovirus vectors for gene transfer, particularly in regard to safety. The authors also discuss rapid advances in other nucleic acid therapeutics such as antisense oligonucleotide drugs and RNA-based therapeutic agents. New gene editing tools that are generating a lot of attention offer the possibility of targeted gene inactivation or insertion of therapeutic genes without the use of viral vectors.

“We are extremely pleased that the Director of the NCATS has chosen our journal to describe his strategic vision for gene 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.

About the Journal
Human Gene Therapy (http://www.liebertpub.com/hgt), 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 (http://www.liebertpub.com/hgt) website.

About the Publisher
Mary Ann Liebert, Inc., publishers (http://www.liebertpub.com/) 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 (http://www.liebertpub.com/) website.