期刊
TRENDS IN MOLECULAR MEDICINE
卷 22, 期 4, 页码 317-327出版社
ELSEVIER SCI LTD
DOI: 10.1016/j.molmed.2016.02.002
关键词
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资金
- Wellcome Trust
- Great Ormond Street Hospital Children's Charity
- National Institute for Health Research Biomedical Research Centre at Great Ormond Street Hospital for Children NHS Foundation Trust and University College London
- Academy of Medical Sciences (AMS) [AMS-SGCL9-Booth] Funding Source: researchfish
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.
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