Combination of engineered FnCas9 and extended gRNAs for PAM-flexible, robust and nucleobase specific editing and diagnostics

Abstract excerpt

<title>Abstract</title> <p>The clinical success of CRISPR therapies is dependent on the safety and efficacy of Cas proteins. The Cas9 from <italic>Francisella novicida</italic> (FnCas9) has negligible affinity for mismatched substrates enabling it to discriminate off-targets in DNA with very high precision even at the level of binding. However, its cellular targeting efficiency is low, limiting its use in therape...

Topics

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• CRISPR-Associated Protein 9
• CRISPR-Cas Systems
• Gene Editing
• HEK293 Cells
• Leber Congenital Amaurosis
• Protein Engineering
• RNA, Guide, CRISPR-Cas Systems
• Streptococcus pyogenes

Identifiers and source

Literature Corpus work

d63f601a-ee61-5da6-a64d-0603cee5bdae

DOI

10.21203/rs.3.rs-3104171/v1