Key Challenges in Bringing CRISPR-Mediated Somatic Cell Therapy into the Clinic

Genome Medicine, 2017

4 Pages Posted: 18 Oct 2017

See all articles by Dianne Nicol

Dianne Nicol

University of Tasmania

Lisa Eckstein

Georgetown University Law Center

Michael Morrison

University of Oxford - Centre for Health, Law and Emerging Technologies

Jacob S. Sherkow

New York Law School; Columbia University - Department of Health Policy and Management; Center for Advanced Studies in Biomedical Innovation Law

Margaret Otlowski

University of Tasmania - Faculty of Law

Tess Whitton

University of Tasmania

Tania Bubela

University of Alberta - Faculty of Public Health

Kathryn Burdon

University of Tasmania

Donald Chalmers

University of Tasmania - Faculty of Law; University of Sydney - Centre for Values, Ethics and the Law in Medicine

Sarah Chan

University of Manchester - Centre for Social Ethics and Policy

Jacqueline Charlesworth

University of Tasmania - Menzies Institute for Medical Research

Christine Critchley

Swinburne University of Technology

Merlin Crossley

University of New South Wales (UNSW)

Sheryl de Lacey

Flinders University

Joanne Dickinson

University of Tasmania

Alex Hewitt

University of Tasmania

Johnathon Liddicoat

University of Cambridge - Faculty of Law; University of Tasmania

Joanne Kamens

Addgene

Kazuto Kato

Osaka, Dept of Biomedical Ethics & Public Policy, Graduate School of Medicine; Kyoto University, Institute for Integrated Cell-Material Sciences

Erica Kleiderman

McGill University

Satoshi Kodama

Kyoto University

David Mackey

The University of Western Australia

Ainsley Newson

The University of Sydney

Jane L Nielsen

University of Tasmania

Jennifer Wagner

Geisinger Health System Inc. - Bioethics Research

Rebekah McWhirter

University of Tasmania

Date Written: September 25, 2017

Abstract

Genome editing using clustered regularly interspersed short palindromic repeats (CRISPR) and CRISPR-associated proteins offers the potential to facilitate safe and effective treatment of genetic diseases refractory to other types of intervention. Here, we identify some of the major challenges for clinicians, regulators, and human research ethics committees in the clinical translation of CRISPR-mediated somatic cell therapy.

Keywords: Genome Editing, CRISPR, Somatic Cell Therapy, Genetic Diseases, Gene Therapy

Suggested Citation

Nicol, Dianne and Eckstein, Lisa and Morrison, Michael and Sherkow, Jacob S. and Otlowski, Margaret and Whitton, Tess and Bubela, Tania and Burdon, Kathryn and Chalmers, Donald and Chan, Sarah and Charlesworth, Jacqueline and Critchley, Christine and Crossley, Merlin and de Lacey, Sheryl and Dickinson, Joanne and Hewitt, Alex and Liddicoat, Johnathon and Kamens, Joanne and Kato, Kazuto and Kleiderman, Erica and Kodama, Satoshi and Mackey, David and Newson, Ainsley and Nielsen, Jane L and Wagner, Jennifer and McWhirter, Rebekah, Key Challenges in Bringing CRISPR-Mediated Somatic Cell Therapy into the Clinic (September 25, 2017). Genome Medicine, 2017. Available at SSRN: https://ssrn.com/abstract=3050477

Dianne Nicol (Contact Author)

University of Tasmania ( email )

French Street
Sandy Bay
Tasmania, 7250
Australia

Lisa Eckstein

Georgetown University Law Center ( email )

600 New Jersey Avenue, NW
Washington, DC 20001
United States

Michael Morrison

University of Oxford - Centre for Health, Law and Emerging Technologies

University of Oxford
Oxford, OX2 7DD
United Kingdom

Jacob S. Sherkow

New York Law School ( email )

185 West Broadway
New York, NY 10013
United States
212.431.2355 (Phone)

HOME PAGE: http://www.nyls.edu/faculty/faculty-profiles/faculty_profiles/jacob-s-sherkow/

Columbia University - Department of Health Policy and Management ( email )

600 West 168th Street, 6th Floor
New York, NY 10032
United States

Center for Advanced Studies in Biomedical Innovation Law ( email )

Studiestraede 6
Studiestrade 6
Copenhagen, DK-1455
Denmark

HOME PAGE: http://jura.ku.dk/cebil/staff/

Margaret Otlowski

University of Tasmania - Faculty of Law ( email )

Private Bag 89
Hobart
Tasmania, 7001
Australia

Tess Whitton

University of Tasmania ( email )

French Street
Sandy Bay
Tasmania, 7250
Australia

Tania Bubela

University of Alberta - Faculty of Public Health ( email )

11405 - 87 Ave.
Edmonton, Alberta T6G 1C9
Canada
(780) 492-9335 (Phone)

Kathryn Burdon

University of Tasmania ( email )

French Street
Sandy Bay
Tasmania, 7250
Australia

Donald Chalmers

University of Tasmania - Faculty of Law ( email )

Private Bag 89
Hobart
Tasmania, 7001
Australia

University of Sydney - Centre for Values, Ethics and the Law in Medicine ( email )

Medical Foundation Building
K25, Level 1 92-94 Parramatta Rd
Camperdown New South Wales, 2006
Australia

Sarah Chan

University of Manchester - Centre for Social Ethics and Policy ( email )

Oxford Road
Manchester, M13 9PL
United Kingdom

Jacqueline Charlesworth

University of Tasmania - Menzies Institute for Medical Research ( email )

Australia

Christine Critchley

Swinburne University of Technology ( email )

Cnr Wakefield and William Streets, Hawthorn Victor
3122 Victoria, Victoria 3122
Australia

Merlin Crossley

University of New South Wales (UNSW) ( email )

Kensington
High St
Sydney, NSW 2052
Australia

Sheryl De Lacey

Flinders University ( email )

GPO Box 2100
Adelaide S.A. 5001, SA 5063
Australia

Joanne Dickinson

University of Tasmania ( email )

French Street
Sandy Bay
Tasmania, 7250
Australia

Alex Hewitt

University of Tasmania ( email )

French Street
Sandy Bay
Tasmania, 7250
Australia

Johnathon Liddicoat

University of Cambridge - Faculty of Law ( email )

10 West Road
Cambridge, CB3 9DZ
United Kingdom

University of Tasmania ( email )

French Street
Sandy Bay
Hobart, Tasmania 7001
Australia

Joanne Kamens

Addgene ( email )

75 Sidney St
Suite 550A
Cambridge, MA 02139
United States

Kazuto Kato

Osaka, Dept of Biomedical Ethics & Public Policy, Graduate School of Medicine

1-3 Machikaneyama-cho, Toyonaka
Osaka, 560-8531
Japan

Kyoto University, Institute for Integrated Cell-Material Sciences

Yoshida-Honmachi
Sakyo-ku
Kyoto, 606-8501
Japan

Erica Kleiderman

McGill University ( email )

1001 Sherbrooke St. W
Montreal, Quebec H3A 1G5
Canada

Satoshi Kodama

Kyoto University ( email )

Yoshida-Honmachi
Sakyo-ku
Kyoto, 606-8501
Japan

David Mackey

The University of Western Australia ( email )

35 Stirling Highway
Crawley, Western Australia 6009
AUSTRALIA

Ainsley Newson

The University of Sydney ( email )

University of Sydney
Sydney, NC NSW 2006
Australia

Jane L Nielsen

University of Tasmania ( email )

French Street
Sandy Bay
Tasmania, 7250
Australia

Jennifer Wagner

Geisinger Health System Inc. - Bioethics Research ( email )

100 North Academy Ave.
Danville, PA 17822-4910
United States

Rebekah McWhirter

University of Tasmania ( email )

French Street
Sandy Bay
Tasmania, 7250
Australia

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