Treatment of monogenic and digenic dominant genetic hearing loss by CRISPR-Cas9 ribonucleoprotein delivery in vivo

Abstract excerpt

<title>Abstract</title> <p>Mutations in Atp2b2, an outer hair cell (OHC) gene, cause dominant hearing loss (HL) in humans. Using a mouse model Atp2b2Obl+, with a dominant HL mutation (Oblivion), we show that liposome-mediated in vivo delivery of CRISPR-Cas9 ribonucleoprotein (RNP) complexes leads to specific editing of the Obl allele. Large deletions encompassing the Obl locus and indels were identified as the re...

Topics

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• Clustered Regularly Interspaced Short Palindromic Repeats
• CRISPR-Cas Systems
• Gene Editing
• Gene Transfer Techniques
• Genetic Therapy
• Hair Cells, Auditory
• Hearing Loss
• Polymorphism, Single Nucleotide

Identifiers and source

Literature Corpus work

50ca0c66-b040-5874-afa6-d00143572c5b

DOI

10.21203/rs.3.rs-1836399/v1