Multi-gene duplication removal in an engineered human cellular MECP2 duplication syndrome model with an IRAK1-MECP2 duplication von Rizvi, Samar Z., Chan, Wing Suen, Maino, Eleonora, Steiman, Sydney, Forguson, Georgiana, Klepfish, Maya, Cohn, Ronald D., Ivakine, Evgueni A.

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Targeted genome editing in vivo corrects a Dmd duplication restoring wild‐type dystrophin expression von Maino, Eleonora, Wojtal, Daria, Evagelou, Sonia L, Farheen, Aiman, Wong, Tatianna W Y, Lindsay, Kyle, Scott, Ori, Rizvi, Samar Z, Hyatt, Elzbieta, Rok, Matthew, Visuvanathan, Shagana, Chiodo, Amanda, Schneeweiss, Michelle, Ivakine, Evgueni A, Cohn, Ronald D

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An Irak1-Mecp2 tandem duplication mouse model for the study of MECP2 duplication syndrome von Maino, Eleonora, Scott, Ori, Rizvi, Samar Z, Chan, Wing Suen, Visuvanathan, Shagana, Zablah, Youssif Ben, Li, Hongbin, Sengar, Ameet S, Salter, Michael W, Jia, Zhengping, Rossant, Janet, Cohn, Ronald D, Gu, Bin, Ivakine, Evgueni A

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Targeted genome editing in vivo corrects a Dmd duplication restoring wild‐type dystrophin expression von Maino, Eleonora, Wojtal, Daria, Evagelou, Sonia L, Farheen, Aiman, Wong, Tatianna W Y, Lindsay, Kyle, Scott, Ori, Rizvi, Samar Z, Hyatt, Elzbieta, Rok, Matthew, Visuvanathan, Shagana, Chiodo, Amanda, Schneeweiss, Michelle, Ivakine, Evgueni A, Cohn, Ronald D

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