ANI Photo | Study sheds more light on advances, challenges in gene therapy for rare diseases

A recent review article Therapy summarises major milestones in the development of gene therapy medical products that have aided in the treatment of a large number of uncommon disorders. The essay also discusses the obstacles to the advancement of gene therapy for rare disorders.
The findings were published in the peer-reviewed journal Human Gene.
Juan Bueren, from Centro de Investigaciones Energeticas Medioambientalies y Tecnologicas (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,” said 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. (ANI)

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