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

12 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

Keywords

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

ASJC Scopus subject areas

Molecular Medicine
Genetics
Cell Biology

Access to Document

10.1016/j.stem.2017.09.007

Fingerprint Dive into the research topics of 'Human Genome Editing in the Clinic: New Challenges in Regulatory Benefit-Risk Assessment'. Together they form a unique fingerprint.

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

@article{8e88787a838a4e01bc3a2a1955e6929c,

title = "Human Genome Editing in the Clinic: New Challenges in Regulatory Benefit-Risk Assessment",

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.",

keywords = "benefit-risk assessment, clinical trials, efficacy, genome editing, off-target effects, regulations, safety",

author = "Mohamed Abou-El-Enein and Toni Cathomen and Zolt{\'a}n Ivics and June, {Carl H.} and Matthias Renner and Schneider, {Christian K.} and Gerhard Bauer",

year = "2017",

month = oct,

day = "5",

doi = "10.1016/j.stem.2017.09.007",

language = "English (US)",

volume = "21",

pages = "427--430",

journal = "Cell Stem Cell",

issn = "1934-5909",

publisher = "Cell Press",

number = "4",

}

TY - JOUR

T1 - Human Genome Editing in the Clinic

T2 - New Challenges in Regulatory Benefit-Risk Assessment

AU - Abou-El-Enein, Mohamed

AU - Cathomen, Toni

AU - Ivics, Zoltán

AU - June, Carl H.

AU - Renner, Matthias

AU - Schneider, Christian K.

AU - Bauer, Gerhard

PY - 2017/10/5

Y1 - 2017/10/5

N2 - 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.

AB - 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.

KW - benefit-risk assessment

KW - clinical trials

KW - efficacy

KW - genome editing

KW - off-target effects

KW - regulations

KW - safety

UR - http://www.scopus.com/inward/record.url?scp=85030462516&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85030462516&partnerID=8YFLogxK

U2 - 10.1016/j.stem.2017.09.007

DO - 10.1016/j.stem.2017.09.007

M3 - Comment/debate

C2 - 28985524

AN - SCOPUS:85030462516

VL - 21

SP - 427

EP - 430

JO - Cell Stem Cell

JF - Cell Stem Cell

SN - 1934-5909

IS - 4

ER -