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

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.

SUBMITTER: Staal FJT 

PROVIDER: S-EPMC6834641 | biostudies-literature | 2019

REPOSITORIES: biostudies-literature