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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

2020Molecular Therapy81 citationsDOIOpen Access PDF

Topics & Concepts

Hair cellHearing lossInner earBiologyGenetic enhancementCochleaMutantGenome editingGeneCell biologyGeneticsMedicineAudiologyCRISPRNeuroscienceHearing, Cochlea, Tinnitus, GeneticsRNA regulation and diseaseRNA and protein synthesis mechanisms
Single and Dual Vector Gene Therapy with AAV9-PHP.B Rescues Hearing in Tmc1 Mutant Mice | Litcius