AUTHOR=Staal Frank J. T. , Aiuti Alessandro , Cavazzana Marina TITLE=Autologous Stem-Cell-Based Gene Therapy for Inherited Disorders: State of the Art and Perspectives JOURNAL=Frontiers in Pediatrics VOLUME=7 YEAR=2019 URL=https://www.frontiersin.org/journals/pediatrics/articles/10.3389/fped.2019.00443 DOI=10.3389/fped.2019.00443 ISSN=2296-2360 ABSTRACT=

Gene therapy using patient's own stem cells is rapidly becoming an alternative to allogeneic stem cell transplantation, especially when suitably compatible donors cannot be found. The advent of efficient virus-based methods for delivering therapeutic genes has enabled the development of genetic medicines for inherited disorders of the immune system, hemoglobinopathies, and a number of devastating metabolic diseases. Here, we briefly review the state of the art in the field, including gene editing approaches. A growing number of pediatric diseases can be successfully cured by hematopoietic stem-cell-based gene therapy.