Treatment of autosomal recessive hearing loss via in vivo CRISPR/Cas9-mediated optimized homology-directed repair in mice

Inner hair cell dysfunction in Klhl18 mutant mice leads to low frequency progressive hearing loss

(2021) Neil J. Ingham et al.   PLoS One

Screened AAV variants permit efficient transduction access to supporting cells and hair cells

(2019) Xinde Hu et al.   Cell Discovery

Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents

(2017) Xue Gao et al.   NATURE

Efficient introduction of specific homozygous and heterozygous mutations using CRISPR/Cas9

(2016) Dominik Paquet et al.   NATURE

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

(2016) Yang Yang et al.   NATURE BIOTECHNOLOGY

Correction of the auditory phenotype in C57BL/6N mice via CRISPR/Cas9-mediated homology directed repair

(2016) Joffrey Mianné et al.   Genome Medicine

Therapeutic genome editing: prospects and challenges

(2015) David Benjamin Turitz Cox et al.   NATURE MEDICINE