Special Issue Features State-of-the-Art in Clinical Gene Therapy
Authors: Roland W. Herzog, Robert M. Frederickson
Journal: Molecular Therapy (2020)

Gene therapy is transforming modern medicine, providing treatments for diseases that were previously untreatable and permanent fixes for diseases for which there was no cure. For instance, gene therapy affords major improvements in the quality of life for patients born with severe genetic disorders, allowing kids to attend school and be active because they are no longer blind or have to live in isolation for fear of infections. In some cases, such as severe neurodegenerative diseases or certain cancers, gene therapy is making the difference between life and death. Gene therapy today represents an important innovation in the pharmaceutical sector, and treatments and cures for a broad array of important diseases are expected to continue to develop according to advanced preclinical and clinical studies. Several startup companies in the area have been launched in the past decades, some of which have been acquired by major pharmaceutical companies. Well over 2,000 clinical gene therapy trials have been conducted to date, which have led to a better understanding of treatment safety and efficacy and better vector and protocol designs. Technology has always driven this field, and it continues to do so. Gene editing technology is the latest to transform gene therapy approaches: CRISPR technology has morphed from promising technology to clinical study within less than a decade and has provided an additional strong momentum to an already accelerating field. The editors are thrilled to call our readers' attention to a special issue of Molecular Therapy designed to showcase the state-of-the-art in clinical gene therapy. The issue will be edited by Jacques P. Tremblay, Andrea Annoni, and Masataka Suzuki and will feature a set of comprehensive articles written by leading experts in clinical gene therapy. These will cover clinical gene therapy based on in vivo and ex vivo gene transfer, clinical applications of oligonucleotide technologies, cancer gene therapy, clinical applications of gene editing, and more. While impressively successful in some areas, clinical gene therapy also faces serious obstacles that impede or complicate treatment, as is the case for other novel types of medicine. Therefore, the issue will include a number of critical commentaries on topics such as high-dose systemic gene delivery, immune and other toxicities, difficult-to-treat diseases such as cystic fibrosis, among others, and also paths to commercialization and questions of the pricing of gene therapy products. Finally, a new type of review article will provide clinical perspectives on the use of approved gene therapy medicines. The diverse articles and commentaries will be authored by some of the leading physician-scientists who are spearheading the translation of gene therapy from the bench to the bedside. In addition, we encourage authors to submit primary research, commentaries, and other opinion pieces for rapid editorial consideration. We aim to publish this special issue of the journal in the first quarter of 2021. When submitting your paper, please select the following category in Editorial Manager: Special Issue – Clinical Gene Therapy. Presubmission enquires are welcome via email to [email protected]