From Genomics to Gene Therapy: Induced Pluripotent Stem Cells Meet Genome Editing

被引：88

作者：

Hotta, Akitsu ^{[1
,2
]}

Yamanaka, Shinya ^{[1
,2
,3
]}

机构：

[1] Kyoto Univ, Ctr iPS Cell Res & Applicat CiRA, Kyoto 6068501, Japan

[2] Kyoto Univ, Inst Integrated Cell Mat Sci iCeMS, Kyoto 6068501, Japan

[3] Gladstone Inst Cardiovasc Dis, San Francisco, CA 94158 USA

来源：

ANNUAL REVIEW OF GENETICS, VOL 49 | 2015年 / 49卷

关键词：

pluripotent stem cells; genetic correction; programmable nuclease; TALEN; CRISPR-Cas9; ZINC-FINGER NUCLEASES; HUMAN IPS CELLS; DNA-BINDING SPECIFICITY; DOUBLE-STRAND BREAKS; HOMOLOGOUS RECOMBINATION; SOMATIC-CELLS; MOUSE EPIBLAST; SELF-RENEWAL; HUMAN ESCS; T-CELLS;

D O I：

10.1146/annurev-genet-112414-054926

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

The advent of induced pluripotent stem (iPS) cells has opened up numerous avenues of opportunity for cell therapy, including the initiation in September 2014 of the first human clinical trial to treat dry age-related macular degeneration. In parallel, advances in genome-editing technologies by site-specific nucleases have dramatically improved our ability to edit endogenous genomic sequences at targeted sites of interest. In fact, clinical trials have already begun to implement this technology to control HIV infection. Genome editing in iPS cells is a powerful tool and enables researchers to investigate the intricacies of the human genome in a dish. In the near future, the groundwork laid by such an approach may expand the possibilities of gene therapy for treating congenital disorders. In this review, we summarize the exciting progress being made in the utilization of genomic editing technologies in pluripotent stem cells and discuss remaining challenges toward gene therapy applications.

引用

页码：47 / 70

页数：24

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