Allele-specific CRISPR-Cas9 editing inactivates a single nucleotide variant associated with collagen VI muscular dystrophy

• Published in: Molecular therapy. Nucleic acids Vol. 35; no. 3; p. 102269
• Main Authors: Bolduc, Véronique, Sizov, Katherine, Brull, Astrid, Esposito, Eric, Chen, Grace S., Uapinyoying, Prech, Sarathy, Apurva, Johnson, Kory R., Bönnemann, Carsten G.
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 10.09.2024; American Society of Gene & Cell Therapy
• Subjects: allele-specific gene editing; collagen VI; CRISPR-Cas9; guide RNA design; MT: RNA/DNA Editing; muscular dystrophy; Original; collagen VI; MT: RNA/DNA Editing; muscular dystrophy; guide RNA design; CRISPR-Cas9; allele-specific gene editing

The application of allele-specific gene editing tools can expand the therapeutic options for dominant genetic conditions, either via gene correction or via allelic gene inactivation in situations where haploinsufficiency is tolerated. Here, we used allele-targeted CRISPR-Cas9 guide RNAs (gRNAs) to i...