Advances and challenges in gene therapy for rare diseases

New Rochelle, NY, November 13, 2023—A new review article in the peer-reviewed journal Human Gene Therapy summarizes the significant milestones in the development of gene therapy medicinal products that have facilitated the treatment of a significant number of rare diseases. The article also describes the challenges in the progress of gene therapy for rare diseases. Click here to read the article now.

Juan Bueren, from Centro de Investigaciones Energéticas Medioambientalies y Tecnológicas (CIEMAT), and President of the European Society for Gene and Cell Therapy (ESGCT), and Alberto Auricchio, from Telethon Institute of Genetics and Medicine (TIGEM), and Vice-President of the European Society for Gene and Cell Therapy, coauthored the article titled “Advances and Challenges in the Development of Gene Therapy Medicinal Products for Rare Diseases.”

“Advances in the generation of integration competent vectors have markedly improved the efficacy and also the safety associated to ex vivo hematopoietic stem cell gene therapies during the last decade,” state the authors. “In addition to ex vivo gene therapy, in vivo gene therapy has shown to be safe and effective therapy in humans. However, also some challenges remain to make this transformative therapeutic approach widely available.”

“Professors Bueren and Auricchio, as the leaders of the ESGCT, provide a uniquely comprehensive perspective on the development of molecular therapies for rare diseases in Europe,” 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.

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. is a global media company dedicated to creating, curating, and delivering impactful peer-reviewed research and authoritative content services to advance the fields of biotechnology and the life sciences, specialized clinical medicine, and public health and policy. For complete information, please visit the Mary Ann Liebert, Inc. website.