METHODS AND COMPOSITIONS FOR THE ADAR-MEDIATED EDITING OF TRANSMEMBRANE CHANNEL-LIKE PROTEIN 1 (TMC1)

摘要

The present invention relates to methods and compositions for editing a TMC1 polynucleotide, e.g., a TMC1 polynucleotide comprising a SNP associated with autosomal recessive non-syndromic hearing loss. The invention also relates to methods and compositions for treating or preventing autosomal recessive non-syndromic hearing loss in a subject.