Treatment of monogenic and digenic dominant genetic hearing loss by CRISPR-Cas9 ribonucleoprotein delivery in vivo von Tao, Yong, Lamas, Veronica, Du, Wan, Zhu, Wenliang, Li, Yiran, Whittaker, Madelynn N., Zuris, John A., Thompson, David B., Rameshbabu, Arun Prabhu, Shu, Yilai, Gao, Xue, Hu, Johnny H., Pei, Charles, Kong, Wei-Jia, Liu, Xuezhong, Wu, Hao, Kleinstiver, Benjamin P., Liu, David R., Chen, Zheng-Yi

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Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants von Walton, Russell T., Christie, Kathleen A., Whittaker, Madelynn N., Kleinstiver, Benjamin P.

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An epigenetic editor to silence genes von Whittaker, Madelynn N, Musunuru, Kiran

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Allele-specific silencing of the gain-of-function mutation in Huntington's disease using CRISPR/Cas9 von Shin, Jun Wan, Hong, Eun Pyo, Park, Seri S, Choi, Doo Eun, Seong, Ihn Sik, Whittaker, Madelynn N, Kleinstiver, Benjamin P, Chen, Richard Z, Lee, Jong-Min

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Efficient in vivo prime editing corrects the most frequent phenylketonuria variant, associated with high unmet medical need von Brooks, Dominique L., Whittaker, Madelynn N., Qu, Ping, Musunuru, Kiran, Ahrens-Nicklas, Rebecca C., Wang, Xiao

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Epigenome Editing Durability Varies Widely Across Cardiovascular Disease Target Genes von Whittaker, Madelynn N, Testa, Lauren C, Quigley, Aidan, Jindal, Ishaan, Cortez-Alvarado, Saúl V, Qu, Ping, Yang, Yifan, Alameh, Mohamad-Gabriel, Musunuru, Kiran, Wang, Xiao

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A base editing strategy using mRNA-LNPs for in vivo correction of the most frequent phenylketonuria variant von Brooks, Dominique L, Whittaker, Madelynn N, Said, Hooda, Dwivedi, Garima, Qu, Ping, Musunuru, Kiran, Ahrens-Nicklas, Rebecca C, Alameh, Mohamad-Gabriel, Wang, Xiao

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Abstract 125: Reversible Epigenome Editing Of PCSK9 As A Therapeutic Strategy von Wang, Xiao, Whittaker, Madelynn N, Jindal, Ishaan, Cortez-Alvarado, Saúl V, Qu, Ping, Musunuru, Kiran

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