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Article

AlPaCas: allele-specific CRISPR gene editing through a protospacer-adjacent-motif (PAM) approach.

Nucleic acids research - 5 Jul 2024

Rosignoli Serena, Lustrino Elisa, Conci Alessio, Fabrizi Alessandra, Rinaldo Serena, Latella Maria Carmela, Enzo Elena, Prosseda Gianni, De Rosa Laura, De Luca Michele, Paiardini Alessandro

Abstract excerpt

Gene therapy of dominantly inherited genetic diseases requires either the selective disruption of the mutant allele or the editing of the specific mutation. The CRISPR-Cas system holds great potential for the genetic correction of single nucleotide variants (SNVs), including dominant mutations. However, distinguishing between single-nucleotide variations in a pathogenic genomic context remains challenging. The...

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Identifiers

PMID: 38795068
DOI: 10.1093/nar/gkae419
PMCID: PMC11223865

Genes

PAM

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

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