Autologous Stem-Cell-Based Gene Therapy for Inherited Disorders: State of the Art and Perspectives

Autologous Stem-Cell-Based Gene Therapy for Inherited Disorders: State of the Art and Perspectives

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...

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Bibliographic Details
Main Authors: Frank J. T. Staal, Alessandro Aiuti, Marina Cavazzana
Format: Article
Language:English
Published: Frontiers Media S.A. 2019-10-01
Series:Frontiers in Pediatrics
Subjects:
gene therapy
SCID
thalassemia
sickle cell disease
gene editing
lysosomal storage disorder
Online Access:https://www.frontiersin.org/article/10.3389/fped.2019.00443/full
Description
Summary: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.
ISSN:2296-2360

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