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Article

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

Molecular therapy : the journal of the American Society of Gene Therapy - 2 May 2018

McMahon Moira A, Prakash Thazha P, Cleveland Don W, Bennett C Frank, Rahdar Meghdad

Abstract excerpt

CRISPR-based gene editing is a powerful technology for engineering mammalian genomes. It holds the potential as a therapeutic, although much-needed in vivo delivery systems have yet to be established. Here, using the Cpf1-crRNA (CRISPR RNA) crystal structure as a guide, we synthesized a series of systematically truncated and chemically modified crRNAs, and identify positions that are amenable to modification...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 29650467
DOI: 10.1016/j.ymthe.2018.02.031
PMCID: PMC5993945

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