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CRISPR to the Rescue: Advances in Gene Editing for the FMR1 Gene

1
The Raymond G. Perelman Center of Cellular and Molecular Therapeutics, Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA
2
Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA
*
Author to whom correspondence should be addressed.
Brain Sci. 2019, 9(1), 17; https://doi.org/10.3390/brainsci9010017
Received: 28 November 2018 / Revised: 13 January 2019 / Accepted: 15 January 2019 / Published: 21 January 2019
(This article belongs to the Special Issue Towards Mechanism-based Treatments for Fragile X Syndrome)
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Abstract

Gene-editing using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) is promising as a potential therapeutic strategy for many genetic disorders. CRISPR-based therapies are already being assessed in clinical trials, and evaluation of this technology in Fragile X syndrome has been performed by a number of groups. The findings from these studies and the advancement of CRISPR-based technologies are insightful as the field continues towards treatments and cures of Fragile X-Associated Disorders (FXADs). In this review, we summarize reports using CRISPR-editing strategies to target Fragile X syndrome (FXS) molecular dysregulation, and highlight how differences in FXS and Fragile X-associated Tremor/Ataxia Syndrome (FXTAS) might alter treatment strategies for each syndrome. We discuss the various modifications and evolutions of the CRISPR toolkit that expand its therapeutic potential, and other considerations for moving these strategies from bench to bedside. The rapidly growing field of CRISPR therapeutics is providing a myriad of approaches to target a gene, pathway, or transcript for modification. As cures for FXADs have remained elusive, CRISPR opens new avenues to pursue. View Full-Text
Keywords: Fragile X syndrome 1; Fragile X-associated Tremor/Ataxia Syndrome 2; CRISPR 3; Trinucleotide Repeat 4; Gene editing Fragile X syndrome 1; Fragile X-associated Tremor/Ataxia Syndrome 2; CRISPR 3; Trinucleotide Repeat 4; Gene editing
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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited (CC BY 4.0).
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Yrigollen, C.M.; Davidson, B.L. CRISPR to the Rescue: Advances in Gene Editing for the FMR1 Gene. Brain Sci. 2019, 9, 17.

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