RNA Based Gene Therapy In A Model Of RNA Based Gene Therapy In A Model Of Usher syndrome

Usher syndrome (USH) is the most prevalent cause of combined deafness and blindness in humans. USH is categorized in three distinct subtypes based on severity of hearing loss and presence of vestibular dysfunction. The hearing loss due to mutations in different USH genes results from disorganization of the hair bundles of the cochlear hair cells. In all three USH subtypes visual impairment is due to retinitis pigmentosa (RP). Mutations in USH2A are the most frequent cause of USH and also recessive forms of RP. In vivo gene edition using programmable nucleases such as CRISPR/Cas9 technology offers a solution to precisely repair the gene instead of delivering a genetically corrected large cDNA molecule.This project is aiming to perform gene repair in an in vitro and an animal model of USH2, by targeting photoreceptors via delivering CRISPR tools via nanoparticles.