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

Mohamed Abou-El-Enein; Toni Cathomen; Zoltán Ivics; Carl H. June; Matthias Renner; Christian K. Schneider; Gerhard Bauer

doi:10.1016/j.stem.2017.09.007

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

Mohamed Abou-El-Enein, Toni Cathomen, Zoltán Ivics, Carl H. June, Matthias Renner, Christian K. Schneider, Gerhard Bauer

Hematology and Oncology

Research output: Contribution to journal › Comment/debate

10 Scopus citations

Abstract

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. 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.

Original language	English (US)
Pages (from-to)	427-430
Number of pages	4
Journal	Cell Stem Cell
Volume	21
Issue number	4
DOIs	https://doi.org/10.1016/j.stem.2017.09.007
State	Published - Oct 5 2017

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Keywords

benefit-risk assessment
clinical trials
efficacy
genome editing
off-target effects
regulations
safety

ASJC Scopus subject areas

Molecular Medicine
Genetics
Cell Biology

Cite this

Abou-El-Enein, M., Cathomen, T., Ivics, Z., June, C. H., Renner, M., Schneider, C. K., & Bauer, G. (2017). Human Genome Editing in the Clinic: New Challenges in Regulatory Benefit-Risk Assessment. Cell Stem Cell, 21(4), 427-430. https://doi.org/10.1016/j.stem.2017.09.007

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10.1016/j.stem.2017.09.007