GeneticseLifePeer-reviewed
Allele-specific gene editing approach for vision loss restoration in RHO-associated Retinitis Pigmentosa
Xiaozhen Liu, Jing Qiao, Ruixuan Jia…·Liping Yang
This paper develops an allele-specific gene editing approach to target the T17M RHO mutant allele in retinitis pigmentosa, showing improved retinal function and photoreceptor preservation in mice.
19 citations