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Review ARTICLE Provisionally accepted The full-text will be published soon. Notify me

Front. Pediatr. | doi: 10.3389/fped.2019.00443

Autologous stem cell-based gene therapy for inherited disorders: state-of-the-art and future prospects

 Frank Staal1*, Alessandro Aiuti2 and  Marina Cavazzana3
  • 1Leiden University Medical Center, Netherlands
  • 2San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Italy
  • 3Necker-Enfants Malades Hospital, France

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.

Keywords: Gene therapy-SCID-Thalassemia-Sickle cell disease-gene editing-lysosomal storage disorders-clinical trial-curative treatment, Gene Therapy, SCID - severe combined immunodeficiency, Thalasemia, gene editing, lysosomal storage disease

Received: 08 Jul 2019; Accepted: 11 Oct 2019.

Copyright: © 2019 Staal, Aiuti and Cavazzana. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

* Correspondence: Prof. Frank Staal, Leiden University Medical Center, Leiden, Netherlands, f.j.t.staal@lumc.nl