CRISPR/Cas9-mediated exon inclusion in Lama2 gene alleviates dystrophic pathology in MDC1A mouse model von Kemaladewi, D, Hyatt, E, Ivakine, Z, Cohn, R

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Gene editing using CRISPR/Cas9 in neuromuscular disorders von Gonorazky, H, Maani, N, Khattak, S, Ivakine, Z, Cohn, R, Dowling, J

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G.O.6 von Yoon, G, Malam, Z, Paton, T, Marshall, C, Hyatt, E, Ivakine, Z, Kemaladewi, D, Forge, C, Lee, K.S, Hawkins, C, Cohn, R.D

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P.347 - CRISPR/Cas9-mediated exon inclusion in Lama2 gene alleviates dystrophic pathology in MDC1A mouse model von Kemaladewi, D., Hyatt, E., Ivakine, Z., Cohn, R.

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P.132 - Gene editing using CRISPR/Cas9 in neuromuscular disorders von Gonorazky, H., Maani, N., Khattak, S., Ivakine, Z., Cohn, R., Dowling, J.

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G.O.6: Lethal disorder of mitochondrial fission caused by mutations in DNM1L von Yoon, G., Malam, Z., Paton, T., Marshall, C., Hyatt, E., Ivakine, Z., Kemaladewi, D., Forge, C., Lee, K.S., Hawkins, C., Cohn, R.D.

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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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Derepression of the IL-10 Gene by CRISPR-Cas Genome Editing as a Therapeutic Strategy for Persistent Immunomodulation of Donor Lungs for Transplantation von Mesaki, K., Juvet, S., Yeung, J., Ivakine, E., Guan, Z., Shathasivam, P., Akhter, A., Wilson, G., Kleinstiver, B., Hu, J., Davidson, A., Cypel, M., Liu, M., Keshavjee, S.

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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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