Innovations Needed for Effective Implementation of Ex Vivo Gene Therapies

Frontiers in Medicine

Marina Cavazzana¹ 

关键词: gene therapy;    hematopoietic stem progenitor cells;    genome-editing technology;    dyserythropoiesis;    hemoglobinopathies;    Wiskott–;    Aldrich syndrome;   

DOI  :  10.3389/fmed.2017.00029

学科分类：医学（综合）

来源: Frontiers

【 摘 要 】

Over the past decade, therapy based on gene addition to hematopoietic stem progenitor cells (HSPCs) has achieved unprecedented curative outcomes for several genetic diseases, such as X-linked severe combined immunodeficiency (X-SCID) (1), adenosine deaminase deficiency (2), metachromatic leukodystrophy (3), hemoglobinopathies (4), and Wiskott–Aldrich syndrome (WAS) (5).

【 授权许可】

CC BY