Norrie Disease: Gene Therapy and Inner Ear Physiology

This lecture covers Norrie disease, a rare genetic disorder affecting the NDP gene and norrin protein. It discusses the disorder's general information, gene therapy design considerations, inner ear anatomy, cochlear function, tonotopical organization, primary auditory pathway, and cochlear hair cells. The lecture also explores CRISPR/Cas9 for targeting mutated alleles and the biological function of norrin in the Wnt signaling pathway.