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=Volume 7 - 2019

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-derived  stem cells is rapidly becoming an alternative to allogeneic stem cell transplantation, especially when suitable compatible  donors cannot be found.  Efficient viral delivery methods for therapeutic genes have allowed the development of genetic medicines for inherited disorders of the immune system, hemoglobinopathies and various devastating metabolic diseases. Here we briefly review the state of the art developments in the field, including gene editing approaches. Concluding, an increasing number of paediatric indications can be successfully cured by hematopoietic stem cell based gene therapy.