CRISPR/Cas9-Mediated Genome Editing for Huntington's Disease.

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State: Public
Version: author
License: Not specified
Serval ID
serval:BIB_49CB978C79C5
Type
Article: article from journal or magazin.
Publication sub-type
Review (review): journal as complete as possible of one specific subject, written based on exhaustive analyses from published work.
Collection
Publications
Institution
Title
CRISPR/Cas9-Mediated Genome Editing for Huntington's Disease.
Journal
Methods in molecular biology
Author(s)
Vachey G., Déglon N.
ISSN
1940-6029 (Electronic)
ISSN-L
1064-3745
Publication state
Published
Issued date
2018
Peer-reviewed
Oui
Volume
1780
Pages
463-481
Language
english
Notes
Publication types: Journal Article
Publication Status: ppublish
Abstract
This chapter describes the potential use of viral-mediated gene transfer in the central nervous system for genome editing in the context of Huntington's disease. Here, we provide protocols that cover the design of various genome editing strategies, the cloning of CRISPR/Cas9 elements into lentiviral vectors, and the assessment of cleavage efficiency, as well as potential unwanted effects.
Keywords
Animals, Brain/pathology, CRISPR-Cas Systems/genetics, Cell Culture Techniques/instrumentation, Cell Culture Techniques/methods, Cloning, Molecular/methods, Disease Models, Animal, Gene Editing/instrumentation, Gene Editing/methods, Genetic Therapy/instrumentation, Genetic Therapy/methods, Genetic Vectors/administration & dosage, Genetic Vectors/genetics, HEK293 Cells, Humans, Huntingtin Protein/genetics, Huntington Disease/genetics, Huntington Disease/pathology, Huntington Disease/therapy, Lentivirus/genetics, Mice, Mice, Transgenic, Mutation, RNA, Guide/genetics, Stereotaxic Techniques/instrumentation, Transfection/instrumentation, Transfection/methods, Brain, CRISPR, Cas9, Genome editing, Huntington’s disease, Lentiviral vector, Stereotaxic surgery
Pubmed
Create date
11/06/2018 9:09
Last modification date
19/06/2020 5:21
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