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

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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
Pages (from-to)	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

Keywords

Biomedical Technology
Cell- and Tissue-Based Therapy/economics
Clinical Medicine/economics
Clustered Regularly Interspaced Short Palindromic Repeats
Humans
Intellectual Property

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

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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), 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.",

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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, pp. 85. https://doi.org/10.1186/s13073-017-0475-4

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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

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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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KW - Cell- and Tissue-Based Therapy/economics

KW - Clinical Medicine/economics

KW - Clustered Regularly Interspaced Short Palindromic Repeats

KW - Humans

KW - Intellectual Property

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DO - 10.1186/s13073-017-0475-4

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VL - 9

SP - 85

JO - Genome medicine

JF - Genome medicine

IS - 1

ER -

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

Abstract

Keywords

UN SDGs

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