Making gene editing a therapeutic reality

This review discusses current bottlenecks in making CRISPR-Cas9-mediated genome editing a therapeutic reality and it outlines recent strategies that aim to overcome these hurdles as well as the scope of current clinical trials that pioneer the medical translation of CRISPR-Cas9. Additionally, this r...

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Detalles Bibliográficos
Autores principales: Conboy, Irina, Murthy, Niren, Etienne, Jessy, Robinson, Zachery
Formato: Online Artículo Texto
Lenguaje:English
Publicado: F1000 Research Limited 2018
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6305220/
https://www.ncbi.nlm.nih.gov/pubmed/30613384
http://dx.doi.org/10.12688/f1000research.16106.1
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author Conboy, Irina
Murthy, Niren
Etienne, Jessy
Robinson, Zachery
author_facet Conboy, Irina
Murthy, Niren
Etienne, Jessy
Robinson, Zachery
author_sort Conboy, Irina
collection PubMed
description This review discusses current bottlenecks in making CRISPR-Cas9-mediated genome editing a therapeutic reality and it outlines recent strategies that aim to overcome these hurdles as well as the scope of current clinical trials that pioneer the medical translation of CRISPR-Cas9. Additionally, this review outlines the specifics of disease-modifying gene editing in recessive versus dominant genetic diseases with the focus on genetic myopathies that are exemplified by Duchenne muscular dystrophy and myotonic dystrophies.
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spelling pubmed-63052202019-01-03 Making gene editing a therapeutic reality Conboy, Irina Murthy, Niren Etienne, Jessy Robinson, Zachery F1000Res Review This review discusses current bottlenecks in making CRISPR-Cas9-mediated genome editing a therapeutic reality and it outlines recent strategies that aim to overcome these hurdles as well as the scope of current clinical trials that pioneer the medical translation of CRISPR-Cas9. Additionally, this review outlines the specifics of disease-modifying gene editing in recessive versus dominant genetic diseases with the focus on genetic myopathies that are exemplified by Duchenne muscular dystrophy and myotonic dystrophies. F1000 Research Limited 2018-12-21 /pmc/articles/PMC6305220/ /pubmed/30613384 http://dx.doi.org/10.12688/f1000research.16106.1 Text en Copyright: © 2018 Conboy I et al. http://creativecommons.org/licenses/by/4.0/ This is an open access article distributed under the terms of the Creative Commons Attribution Licence, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Review
Conboy, Irina
Murthy, Niren
Etienne, Jessy
Robinson, Zachery
Making gene editing a therapeutic reality
title Making gene editing a therapeutic reality
title_full Making gene editing a therapeutic reality
title_fullStr Making gene editing a therapeutic reality
title_full_unstemmed Making gene editing a therapeutic reality
title_short Making gene editing a therapeutic reality
title_sort making gene editing a therapeutic reality
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6305220/
https://www.ncbi.nlm.nih.gov/pubmed/30613384
http://dx.doi.org/10.12688/f1000research.16106.1
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