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...

Full description

Saved in:
Bibliographic Details
Published in:Frontiers in pediatrics 2019-10, Vol.7, p.443-443
Main Authors: Staal, Frank J. T., Aiuti, Alessandro, Cavazzana, Marina
Format: Article
Language:English
Subjects:
gene editing
gene therapy
lysosomal storage disorder
Pediatrics
SCID
sickle cell disease
thalassemia
Citations: Items that this one cites
Items that cite this one
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
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
2296-2360
DOI:10.3389/fped.2019.00443