Genome editing offers hope for genetic hearing loss

New therapy developed to repair GJB2 R75W mutation

Scientists from Juntendo University and The University of Tokyo have created an adeno-associated virus (AAV)-based genome editing approach to treat genetic hearing loss caused by the GJB2 R75W mutation. Their findings, published in JCI Insight, demonstrate the potential of this innovative therapy.

Hearing loss, when caused by the GJB2 gene mutation, results from fragmented gap junction plaques affecting auditory function.

While recessive mutations can be treated via gene replacement, dominant-negative mutations like R75W require genome…