Engineered PAM-flexible FnCas9 variants for robust and specific genome editing and diagnostics

Abstract excerpt

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

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

78bf09a1-ce77-5ac5-8dcf-f072985d7b3d

DOI

10.21203/rs.3.rs-990232/v2