Gene therapy using haematopoietic stem and progenitor cells

被引：173

作者：

Ferrari, Giuliana ^{[1
,2
]}

Thrasher, Adrian J. ^{[3
,4
]}

Aiuti, Alessandro ^{[1
,2
,5
]}

机构：

[1] Univ Vita Salute San Raffaele, Milan, Italy

[2] IRCCS San Raffaele Sci Inst, San Raffaele Telethon Inst Gene Therapy, Milan, Italy

[3] UCL Great Ormond St Inst Child Hlth, Mol & Cellular Immunol Sect, London, England

[4] Great Ormond St Hosp Children NHS Fdn Trust, London, England

[5] IRCCS San Raffaele Sci Inst, Pediat Immunohematol Unit, Milan, Italy

来源：

NATURE REVIEWS GENETICS | 2021年 / 22卷 / 04期

基金：

英国惠康基金;

关键词：

SEVERE COMBINED IMMUNODEFICIENCY; BONE-MARROW-TRANSPLANTATION; X-LINKED ADRENOLEUKODYSTROPHY; CHRONIC GRANULOMATOUS-DISEASE; RETROVIRAL INTEGRATION SITES; IN-VIVO; BETA-THALASSEMIA; CLONAL TRACKING; BLOOD-CELLS; RECONSTITUTION;

D O I：

10.1038/s41576-020-00298-5

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

Haematopoietic stem and progenitor cell (HSPC) gene therapy has emerged as an effective treatment modality for monogenic disorders of the blood system such as primary immunodeficiencies and beta-thalassaemia. Medicinal products based on autologous HSPCs corrected using lentiviral and gammaretroviral vectors have now been approved for clinical use, and the site-specific genome modification of HSPCs using gene editing techniques such as CRISPR-Cas9 has shown great clinical promise. Preclinical studies have shown engineered HSPCs could also be used to cross-correct non-haematopoietic cells in neurodegenerative metabolic diseases. Here, we review the most recent advances in HSPC gene therapy and discuss emerging strategies for using HSPC gene therapy for a range of diseases.

引用

页码：216 / 234

页数：19

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