Engineering precision therapies: lessons and motivations from the clinic

被引：6

作者：

Xie, Mingqi ^{[1
,2
,3
]}

Viviani, Mirta ^{[1
,2
,3
]}

Fussenegger, Martin ^{[4
,5
]}

机构：

[1] Westlake Univ, Sch Life Sci, Key Lab Growth Regulat & Translat Res Zhejiang Pr, Hangzhou 310024, Zhejiang, Peoples R China

[2] Westlake Lab Life Sci & Biomed, Hangzhou 310024, Zhejiang, Peoples R China

[3] Westlake Inst Adv Study, Inst Basic Med Sci, Hangzhou 310024, Zhejiang, Peoples R China

[4] Swiss Fed Inst Technol, Dept Biosyst Sci & Engn, Basel, Switzerland

[5] Univ Basel, Fac Sci, Basel, Switzerland

来源：

SYNTHETIC BIOLOGY | 2020年 / 6卷 / 01期

基金：

欧洲研究理事会;

关键词：

translational medicine; synthetic biology; cell-based therapies; gene therapy; ATMP; CHIMERIC-ANTIGEN-RECEPTOR; T-CELL THERAPY; RETROVIRAL REPLICATING VECTOR; GENE-THERAPY; SYNTHETIC BIOLOGY; ONCOLYTIC ADENOVIRUS; ANTITUMOR EFFICACY; MOUSE MODEL; STEM-CELLS; BETA-HEMOGLOBINOPATHIES;

D O I：

10.1093/synbio/ysaa024

中图分类号：

Q5 [生物化学];

学科分类号：

071010 ; 081704 ;

摘要：

In the past decade, gene- and cell-based therapies have been at the forefront of the biomedical revolution. Synthetic biology, the engineering discipline of building sophisticated 'genetic software' to enable precise regulation of gene activities in living cells, has been a decisive success factor of these new therapies. Here, we discuss the core technologies and treatment strategies that have already gained approval for therapeutic applications in humans. We also review promising preclinical work that could either enhance the efficacy of existing treatment strategies or pave the way for new precision medicines to treat currently intractable human conditions.

引用

页数：21

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