Treating Immunodeficiency through HSC Gene Therapy

被引：78

作者：

Booth, Claire ^{[1
,2
]}

Gaspar, H. Bobby ^{[1
,2
]}

Thrasher, Adrian J. ^{[1
,2
]}

机构：

[1] UCL Inst Child Hlth, Mol & Cellular Immunol Sect, London, England

[2] Great Ormond St Hosp Sick Children, Dept Paediat Immunol, London, England

来源：

TRENDS IN MOLECULAR MEDICINE | 2016年 / 22卷 / 04期

基金：

英国惠康基金;

关键词：

CHRONIC GRANULOMATOUS-DISEASE; LEUKOCYTE ADHESION DEFICIENCY; HEMATOPOIETIC STEM-CELLS; ADENOSINE-DEAMINASE-DEFICIENCY; WISKOTT-ALDRICH SYNDROME; LENTIVIRAL VECTOR; LYMPHOPROLIFERATIVE DISEASE; MURINE MODEL; X-CGD; MICE;

D O I：

10.1016/j.molmed.2016.02.002

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

Haematopoietic stem cell (HSC) gene therapy has been successfully employed as a therapeutic option to treat specific inherited immune deficiencies, including severe combined immune deficiencies (SCID) over the past two decades. Initial clinical trials using first-generation gamma-retroviral vectors to transfer corrective DNA demonstrated clinical benefit for patients, but were associated with leukemogenesis in a number of cases. Safer vectors have since been developed, affording comparable efficacy with an improved biosafety profile. These vectors are now in Phase I/II clinical trials for a number of immune disorders with more preclinical studies underway. Targeted gene editing allowing precise DNA correction via platforms such as ZFNs, TALENs and CRISPR/Cas9 may now offer promising strategies to improve the safety and efficacy of gene therapy in the future.

引用

页码：317 / 327

页数：11

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