Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD von Wallace, Lindsay M, Saad, Nizar Y, Pyne, Nettie K, Fowler, Allison M, Eidahl, Jocelyn O, Domire, Jacqueline S, Griffin, Danielle A, Herman, Adam C, Sahenk, Zarife, Rodino-Klapac, Louise R, Harper, Scott Q

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Aberrant splicing in transgenes containing introns, exons, and V5 epitopes: lessons from developing an FSHD mouse model expressing a D4Z4 repeat with flanking genomic sequences von Ansseau, Eugénie, Domire, Jacqueline S, Wallace, Lindsay M, Eidahl, Jocelyn O, Guckes, Susan M, Giesige, Carlee R, Pyne, Nettie K, Belayew, Alexandra, Harper, Scott Q

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Allele-specific RNA interference prevents neuropathy in Charcot-Marie-Tooth disease type 2D mouse models von Morelli, Kathryn H, Griffin, Laurie B, Pyne, Nettie K, Wallace, Lindsay M, Fowler, Allison M, Oprescu, Stephanie N, Takase, Ryuichi, Wei, Na, Meyer-Schuman, Rebecca, Mellacheruvu, Dattatreya, Kitzman, Jacob O, Kocen, Samuel G, Hines, Timothy J, Spaulding, Emily L, Lupski, James R, Nesvizhskii, Alexey, Mancias, Pedro, Butler, Ian J, Yang, Xiang-Lei, Hou, Ya-Ming, Antonellis, Anthony, Harper, Scott Q, Burgess, Robert W

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Evaluation of safety and early efficacy of AAV gene therapy in mouse models of vanishing white matter disease von Herstine, Jessica A., Chang, Pi-Kai, Chornyy, Sergiy, Stevenson, Tamara J., Sunshine, Alex C., Nokhrina, Ksenia, Rediger, Jessica, Wentz, Julia, Vetter, Tatyana A., Scholl, Erika, Holaway, Caleb, Pyne, Nettie K., Bratasz, Anna, Yeoh, Stewart, Flanigan, Kevin M., Bonkowsky, Joshua L., Bradbury, Allison M.

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RNAi-Based Gene Therapy Rescues Developmental and Epileptic Encephalopathy in a Genetic Mouse Model von Aimiuwu, Osasumwen V., Fowler, Allison M., Sah, Megha, Teoh, Jia Jie, Kanber, Ayla, Pyne, Nettie K., Petri, Sabrina, Rosenthal-Weiss, Chana, Yang, Mu, Harper, Scott Q., Frankel, Wayne N.

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AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD von Giesige, Carlee R, Wallace, Lindsay M, Heller, Kristin N, Eidahl, Jocelyn O, Saad, Nizar Y, Fowler, Allison M, Pyne, Nettie K, Al-Kharsan, Mustafa, Rashnonejad, Afrooz, Chermahini, Gholamhossein Amini, Domire, Jacqueline S, Mukweyi, Diana, Garwick-Coppens, Sara E, Guckes, Susan M, McLaughlin, K John, Meyer, Kathrin, Rodino-Klapac, Louise R, Harper, Scott Q

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Aberrant Splicing in Transgenes Containing Introns, Exons, and V5 Epitopes: Lessons from Developing an FSHD Mouse Model Expressing a D4Z4 Repeat with Flanking Genomic Sequences: e0... von Ansseau, Eugenie, Domire, Jacqueline S, Wallace, Lindsay M, Eidahl, Jocelyn O, Guckes, Susan M, Giesige, Carlee R, Pyne, Nettie K, Belayew, Alexandra, Harper, Scott Q

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