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

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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Bibliographic Details
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

Internet

https://www.frontiersin.org/articles/10.3389/fnins.2021.632522/full

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