In vivo Genome Editing Therapeutic Approaches for Neurological Disorders: Where Are We in the Translational Pipeline?

In vivo genome editing tools, such as those based on CRISPR, have been increasingly utilized in both basic and translational neuroscience research. There are currently nine in vivo non-CNS genome editing therapies in clinical trials, and the pre-clinical pipeline of major biotechnology companies dem...

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Main Authors:	Pablo Lubroth, Gaia Colasante, Gabriele Lignani
Format:	Article
Language:	English
Published:	Frontiers Media S.A. 2021-02-01
Series:	Frontiers in Neuroscience
Subjects:	genome editing neurological disease CRISPR (clustered regularly interspaced short palindromic repeat)/Cas9 (CRISPR associated protein 9)-mediated genome editing biotech companies translational pipeline
Online Access:	https://www.frontiersin.org/articles/10.3389/fnins.2021.632522/full

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spelling	doaj-2063eeb5300f4db198063412b3ef66822021-02-18T09:06:20ZengFrontiers Media S.A.Frontiers in Neuroscience1662-453X2021-02-011510.3389/fnins.2021.632522632522In vivo Genome Editing Therapeutic Approaches for Neurological Disorders: Where Are We in the Translational Pipeline?Pablo Lubroth0Gaia Colasante1Gabriele Lignani2Hummingbird Ventures, London, United KingdomStem Cell and Neurogenesis Unit, Division of Neuroscience, Ospedale San Raffaele, Milan, ItalyDepartment of Clinical and Experimental Epilepsy, UCL Queen Square Institute of Neurology, University College London, London, United KingdomIn vivo genome editing tools, such as those based on CRISPR, have been increasingly utilized in both basic and translational neuroscience research. There are currently nine in vivo non-CNS genome editing therapies in clinical trials, and the pre-clinical pipeline of major biotechnology companies demonstrate that this number will continue to grow. Several biotechnology companies commercializing in vivo genome editing and modification technologies are developing therapies for CNS disorders with accompanying large partnering deals. In this review, the authors discuss the current genome editing and modification therapy pipeline and those in development to treat CNS disorders. The authors also discuss the technical and commercial limitations to translation of these same therapies and potential avenues to overcome these hurdles.https://www.frontiersin.org/articles/10.3389/fnins.2021.632522/fullgenome editingneurological diseaseCRISPR (clustered regularly interspaced short palindromic repeat)/Cas9 (CRISPR associated protein 9)-mediated genome editingbiotech companiestranslational pipeline
collection	DOAJ
language	English
format	Article
sources	DOAJ
author	Pablo Lubroth Gaia Colasante Gabriele Lignani
spellingShingle	Pablo Lubroth Gaia Colasante Gabriele Lignani In vivo Genome Editing Therapeutic Approaches for Neurological Disorders: Where Are We in the Translational Pipeline? Frontiers in Neuroscience genome editing neurological disease CRISPR (clustered regularly interspaced short palindromic repeat)/Cas9 (CRISPR associated protein 9)-mediated genome editing biotech companies translational pipeline
author_facet	Pablo Lubroth Gaia Colasante Gabriele Lignani
author_sort	Pablo Lubroth
title	In vivo Genome Editing Therapeutic Approaches for Neurological Disorders: Where Are We in the Translational Pipeline?
title_short	In vivo Genome Editing Therapeutic Approaches for Neurological Disorders: Where Are We in the Translational Pipeline?
title_full	In vivo Genome Editing Therapeutic Approaches for Neurological Disorders: Where Are We in the Translational Pipeline?
title_fullStr	In vivo Genome Editing Therapeutic Approaches for Neurological Disorders: Where Are We in the Translational Pipeline?
title_full_unstemmed	In vivo Genome Editing Therapeutic Approaches for Neurological Disorders: Where Are We in the Translational Pipeline?
title_sort	in vivo genome editing therapeutic approaches for neurological disorders: where are we in the translational pipeline?
publisher	Frontiers Media S.A.
series	Frontiers in Neuroscience
issn	1662-453X
publishDate	2021-02-01
description	In vivo genome editing tools, such as those based on CRISPR, have been increasingly utilized in both basic and translational neuroscience research. There are currently nine in vivo non-CNS genome editing therapies in clinical trials, and the pre-clinical pipeline of major biotechnology companies demonstrate that this number will continue to grow. Several biotechnology companies commercializing in vivo genome editing and modification technologies are developing therapies for CNS disorders with accompanying large partnering deals. In this review, the authors discuss the current genome editing and modification therapy pipeline and those in development to treat CNS disorders. The authors also discuss the technical and commercial limitations to translation of these same therapies and potential avenues to overcome these hurdles.
topic	genome editing neurological disease CRISPR (clustered regularly interspaced short palindromic repeat)/Cas9 (CRISPR associated protein 9)-mediated genome editing biotech companies translational pipeline
url	https://www.frontiersin.org/articles/10.3389/fnins.2021.632522/full
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In vivo Genome Editing Therapeutic Approaches for Neurological Disorders: Where Are We in the Translational Pipeline?

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