Human Genome Editing in the Clinic: New Challenges in Regulatory Benefit-Risk Assessment

被引：26

作者：

Abou-El-Enein, Mohamed ^{[1
]}

Cathomen, Toni ^{[2
,3
]}

Ivics, Zoltan ^{[4
]}

June, Carl H. ^{[5
]}

Renner, Matthias ^{[4
]}

Schneider, Christian K. ^{[6
]}

Bauer, Gerhard ^{[7
]}

机构：

[1] Charite Univ Med Berlin, Berlin Brandenburg Ctr Regenerat Therapies BCRT, Berlin, Germany

[2] Univ Freiburg, Med Ctr, Inst Transfus Med & Gene Therapy, Freiburg, Germany

[3] Univ Freiburg, Fac Med, Freiburg, Germany

[4] Paul Ehrlich Inst, Div Med Biotechnol, Langen, Germany

[5] Univ Penn, Ctr Cellular Therapies, Perelman Sch Med, Philadelphia, PA 19104 USA

[6] Med & Healthcare Prod Regulatory Agcy, NIBSC, S Mimms, Herts, England

[7] Univ Calif Davis, IRC, Sacramento, CA 95817 USA

来源：

CELL STEM CELL | 2017年 / 21卷 / 04期

关键词：

TECHNOLOGIES; NUCLEASES;

D O I：

10.1016/j.stem.2017.09.007

中图分类号：

Q813 [细胞工程];

学科分类号：

摘要：

As genome editing rapidly progresses toward the realization of its clinical promise, assessing the suitability of current tools and processes used for its benefit-risk assessment is critical. Although current regulations may initially provide an adequate regulatory framework, improvements are recommended to overcome several existing technology-based safety and efficacy issues.

引用

页码：427 / 430

页数：4

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