Generation of marmoset monkeys with a non-mosaic disruption of the OTOF gene as a model of human deafness - PubMed
5 hours ago
- #non-human primate model
- #gene therapy
- #auditory synaptopathy
- Generated marmoset monkeys with non-mosaic OTOF gene disruption to model human deafness.
- Used CRISPR-Cas9 on zygotes to create biallelic OTOF knockout marmosets born and raised normally.
- Confirmed profound auditory synaptopathy via brainstem recordings and lack of otoferlin in inner hair cells.
- Model supports preclinical optimization of OTOF gene therapy for pediatric clinical trials.