Methods and techniques enabling multi-kilobase long-range genomic rewrite/replace editing

Abstract excerpt

CRISPR enabled cell and gene therapies have the potential to revolutionize the field of genetic medicine. However, the vast majority of rare diseases remain untreatable due to the limitations of current tools and techniques. To date, most corrective therapeutic approaches have been restricted to mutation-by-mutation approaches, where either HDR, or newer techniques such as base or prime editing, rewrite small regi...

Topics

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• CRISPR-Associated Protein 9
• CRISPR-Cas Systems
• DNA Breaks, Double-Stranded
• Gene Editing
• Genetic Therapy
• Mammals
• Mutation
• Phenotype

Identifiers and source

Literature Corpus work

f3253c0d-5f6c-5615-b997-0a19263b2345

DOI

10.1101/2023.08.05.551844