Key challenges in bringing CRISPR-mediated somatic cell therapy into the clinic

Dianne Nicol; Lisa Eckstein; Michael Morrison; Jacob S. Sherkow; Margaret Otlowski; Tess Whitton; Tania Bubela; Kathryn P. Burdon; Don Chalmers; Sarah Chan; Jac Charlesworth; Christine Critchley; Merlin Crossley; Sheryl de Lacey; Joanne L. Dickinson; Alex W. Hewitt; Joanne Kamens; Kazuto Kato; Erika Kleiderman; Satoshi Kodama; John Liddicoat; David A. Mackey; Ainsley J. Newson; Jane Nielsen; Jennifer K. Wagner; Rebekah E. McWhirter

doi:10.1186/s13073-017-0475-4

Key challenges in bringing CRISPR-mediated somatic cell therapy into the clinic

Dianne Nicol, Lisa Eckstein, Michael Morrison, Jacob S. Sherkow, Margaret Otlowski, Tess Whitton, Tania Bubela, Kathryn P. Burdon, Don Chalmers, Sarah Chan, Jac Charlesworth, Christine Critchley, Merlin Crossley, Sheryl de Lacey, Joanne L. Dickinson, Alex W. Hewitt, Joanne Kamens, Kazuto Kato, Erika Kleiderman, Satoshi KodamaJohn Liddicoat, David A. Mackey, Ainsley J. Newson, Jane Nielsen, Jennifer K. Wagner, Rebekah E. McWhirter

Centre for Ophthalmology and Visual Science (affiliated with the Lions Eye Institute)

Research output: Contribution to journal › Comment/debate › peer-review

15 Citations (Scopus)

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.

Original language	English
Article number	85
Journal	Genome Medicine
Volume	9
Issue number	1
DOIs	https://doi.org/10.1186/s13073-017-0475-4
Publication status	Published - 25 Sept 2017

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Nicol, D., Eckstein, L., Morrison, M., Sherkow, J. S., Otlowski, M., Whitton, T., Bubela, T., Burdon, K. P., Chalmers, D., Chan, S., Charlesworth, J., Critchley, C., Crossley, M., de Lacey, S., Dickinson, J. L., Hewitt, A. W., Kamens, J., Kato, K., Kleiderman, E., ... McWhirter, R. E. (2017). Key challenges in bringing CRISPR-mediated somatic cell therapy into the clinic. Genome Medicine, 9(1), Article 85. https://doi.org/10.1186/s13073-017-0475-4

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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.",

author = "Dianne Nicol and Lisa Eckstein and Michael Morrison and Sherkow, {Jacob S.} and Margaret Otlowski and Tess Whitton and Tania Bubela and Burdon, {Kathryn P.} and Don Chalmers and Sarah Chan and Jac Charlesworth and Christine Critchley and Merlin Crossley and {de Lacey}, Sheryl and Dickinson, {Joanne L.} and Hewitt, {Alex W.} and Joanne Kamens and Kazuto Kato and Erika Kleiderman and Satoshi Kodama and John Liddicoat and Mackey, {David A.} and Newson, {Ainsley J.} and Jane Nielsen and Wagner, {Jennifer K.} and McWhirter, {Rebekah E.}",

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Nicol, D, Eckstein, L, Morrison, M, Sherkow, JS, Otlowski, M, Whitton, T, Bubela, T, Burdon, KP, Chalmers, D, Chan, S, Charlesworth, J, Critchley, C, Crossley, M, de Lacey, S, Dickinson, JL, Hewitt, AW, Kamens, J, Kato, K, Kleiderman, E, Kodama, S, Liddicoat, J, Mackey, DA, Newson, AJ, Nielsen, J, Wagner, JK & McWhirter, RE 2017, 'Key challenges in bringing CRISPR-mediated somatic cell therapy into the clinic', Genome Medicine, vol. 9, no. 1, 85. https://doi.org/10.1186/s13073-017-0475-4

TY - JOUR

T1 - Key challenges in bringing CRISPR-mediated somatic cell therapy into the clinic

AU - Nicol, Dianne

AU - Eckstein, Lisa

AU - Morrison, Michael

AU - Sherkow, Jacob S.

AU - Otlowski, Margaret

AU - Whitton, Tess

AU - Bubela, Tania

AU - Burdon, Kathryn P.

AU - Chalmers, Don

AU - Chan, Sarah

AU - Charlesworth, Jac

AU - Critchley, Christine

AU - Crossley, Merlin

AU - de Lacey, Sheryl

AU - Dickinson, Joanne L.

AU - Hewitt, Alex W.

AU - Kamens, Joanne

AU - Kato, Kazuto

AU - Kleiderman, Erika

AU - Kodama, Satoshi

AU - Liddicoat, John

AU - Mackey, David A.

AU - Newson, Ainsley J.

AU - Nielsen, Jane

AU - Wagner, Jennifer K.

AU - McWhirter, Rebekah E.

PY - 2017/9/25

Y1 - 2017/9/25

N2 - 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.

AB - 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.

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U2 - 10.1186/s13073-017-0475-4

DO - 10.1186/s13073-017-0475-4

M3 - Comment/debate

C2 - 28946923

AN - SCOPUS:85029832036

SN - 1756-994X

VL - 9

JO - Genome Medicine

JF - Genome Medicine

IS - 1

M1 - 85

ER -

Key challenges in bringing CRISPR-mediated somatic cell therapy into the clinic

Abstract

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