CRISPR/Cas9-Mediated Genome Editing for Huntington's Disease.
Methods in molecular biology (Clifton, N.J.), 1780: 463-481 PMID 29856031 DOI 10.1007/978-1-4939-7825-0_21
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.