Single and Dual Vector Gene Therapy with AAV9-PHP.B Rescues Hearing in Tmc1 Mutant Mice
Jason Wu, Paola Solanes, Carl Nist-Lund, Sofia Spataro, Olga Shubina-Oleinik, Irina Marcovich, Hannah Goldberg, Bernard L. Schneider, Jeffrey R. Holt
Topics & Concepts
Hair cellHearing lossInner earBiologyGenetic enhancementCochleaMutantGenome editingGeneCell biologyGeneticsMedicineAudiologyCRISPRNeuroscienceHearing, Cochlea, Tinnitus, GeneticsRNA regulation and diseaseRNA and protein synthesis mechanisms