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Gene therapy: Gene-editing therapy for neurological disease.

作者信息

McMahon Moira A, Cleveland Don W

机构信息

Ludwig Institute and Department of Cellular and Molecular Medicine, University of California, San Diego, 9500 Gilman Drive, La Jolla, California 92093-0670, USA.

出版信息

Nat Rev Neurol. 2017 Jan;13(1):7-9. doi: 10.1038/nrneurol.2016.190. Epub 2016 Dec 16.

DOI:10.1038/nrneurol.2016.190

PMID:27982043

Abstract

摘要

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Gene therapy: Gene-editing therapy for neurological disease.

Nat Rev Neurol. 2017 Jan;13(1):7-9. doi: 10.1038/nrneurol.2016.190. Epub 2016 Dec 16.

Huntington disease: Selective deactivation of Huntington disease mutant allele by CRISPR-Cas9 gene editing.

Nat Rev Neurol. 2016 Nov;12(11):614-615. doi: 10.1038/nrneurol.2016.151. Epub 2016 Sep 30.

CRISPR takes on Huntington's disease.

Nature. 2018 May;557(7707):S42-S43. doi: 10.1038/d41586-018-05177-y.

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Rev Neurol (Paris). 2018 Sep-Oct;174(7-8):487-488. doi: 10.1016/j.neurol.2018.04.006. Epub 2018 May 17.

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J Cent Nerv Syst Dis. 2018 Jul 10;10:1179573518787469. doi: 10.1177/1179573518787469. eCollection 2018.

Chemically Modified Cpf1-CRISPR RNAs Mediate Efficient Genome Editing in Mammalian Cells.

Mol Ther. 2018 May 2;26(5):1228-1240. doi: 10.1016/j.ymthe.2018.02.031. Epub 2018 Mar 6.

Close encounters: Moving along bumps, breaks, and bubbles on expanded trinucleotide tracts.

DNA Repair (Amst). 2017 Aug;56:144-155. doi: 10.1016/j.dnarep.2017.06.017. Epub 2017 Jun 9.

本文引用的文献

Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR/Cas9.

Hum Mol Genet. 2016 Oct 15;25(20):4566-4576. doi: 10.1093/hmg/ddw286.

Reactivation of FMR1 by CRISPR/Cas9-Mediated Deletion of the Expanded CGG-Repeat of the Fragile X Chromosome.

PLoS One. 2016 Oct 21;11(10):e0165499. doi: 10.1371/journal.pone.0165499. eCollection 2016.

Synthetic CRISPR RNA-Cas9-guided genome editing in human cells.

Proc Natl Acad Sci U S A. 2015 Dec 22;112(51):E7110-7. doi: 10.1073/pnas.1520883112. Epub 2015 Nov 16.

Reversion of FMR1 Methylation and Silencing by Editing the Triplet Repeats in Fragile X iPSC-Derived Neurons.

Cell Rep. 2015 Oct 13;13(2):234-41. doi: 10.1016/j.celrep.2015.08.084. Epub 2015 Oct 1.

Therapeutic genome editing: prospects and challenges.

Nat Med. 2015 Feb;21(2):121-31. doi: 10.1038/nm.3793.

Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS.

Mol Ther. 2013 Dec;21(12):2148-59. doi: 10.1038/mt.2013.211. Epub 2013 Sep 6.

Multiplex genome engineering using CRISPR/Cas systems.

Science. 2013 Feb 15;339(6121):819-23. doi: 10.1126/science.1231143. Epub 2013 Jan 3.

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J Clin Invest. 2006 Aug;116(8):2290-6. doi: 10.1172/JCI25424. Epub 2006 Jul 27.

Gene therapy: Gene-editing therapy for neurological disease.

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