Gene editing with adeno-associated virus vector offers hope for hereditary deafness

Gene editing with adeno-associated virus vector offers hope for hereditary deafness

An R75W mutation in the gap junction β2 (GJB2) gene causes severe fragmentation of gap junction plaques, connecting adjacent cells and leading to syndromic hearing loss. In a new experimental study, scientists from Juntendo University in collaboration with researchers from the University of Tokyo have developed an adeno-associated virus (AAV) vector-mediated genome editing approach to repair the R75W mutation.

Experts Discuss Guardrails for Heritable Human Genome Editing

Around 2018–19, there was not a bigger science and ethical story than the debate over heritable human genome editing (HHGE) and the scandal over the “CRISPR babies.” The scientist, He Jiankui, who attempted to engineer the germline of human embryos, resulting in the birth of twin girls in late 2018, was later jailed by Chinese authorities. Even as groups of leading scientists called for a temporary moratorium, several distinguished scientific or…

Genome editing offers hope for genetic hearing loss

New therapy developed to repair GJB2 R75W mutation