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

Frontiers in Pediatrics

article

Frank J. T. Staal¹  Alessandro Aiuti²  Marina Cavazzana⁴ 

[1] Department of Immunohematology and Blood Transfusion (IHB), Leiden University Medical Center;Paediatric Immunohematology Unit, San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute;San Raffaele University;Biotherapy Department, Necker Children's Hospital

关键词: gene therapy;    SCID;    thalassemia;    sickle cell disease;    gene editing;    lysosomal storage disorder;    clinical trial;    curative treatment;   

DOI  :  10.3389/fped.2019.00443

学科分类：社会科学、人文和艺术（综合）

来源: Frontiers

【 摘 要 】

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.

【 授权许可】

CC BY