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SaveA novel mouse model of Duchenne muscular dystrophy carrying a multi-exonic Dmd deletion exhibits progressive muscular dystrophy and early-onset cardiomyopathy (vol 13, dmm045369, 2020)
Wai, Tatianna; Wong, Ying; Ahmed, Abdalla; Yang, Grace; Maino, Eleonora; Steiman, Sydney; Hyatt, Elzbieta; Chan, Parry; Lindsay, Kyle; Wong, Nicole; Golebiowski, Diane; Schneider, Joel; Delgado-Olguin, Paul; Ivakine, Evgueni A.; Cohn, Ronald D. Share Save Share Save Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophyBirch, Sharla M.; Lawlor, Michael W.; Conlon, Thomas J.; Guo, Lee-Jae; Crudele, Julie M.; Hawkins, Eleanor C.; Nghiem, Peter P.; Ahn, Mihye; Meng, Hui; Beatka, Margaret J.; Fickau, Brittany A.; Prieto, Juan C.; Styner, Martin A.; Struharik, Michael J.; Shanks, Courtney; Brown, Kristy J.; Golebiowski, Diane; Bettis, Amanda K.; Balog-Alvarez, Cynthia J.; Clement, Nathalie; Coleman, Kirsten E.; Corti, Manuela; Pan, Xiufang; Hauschka, Stephen D.; Gonzalez, J. Patrick; Morris, Carl A.; Schneider, Joel S.; Duan, Dongsheng; Chamberlain, Jeffrey S.; Byrne, Barry J.; Kornegay, Joe. N. Share Save Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff MiceLahey, Hannah G.; Webber, Chelsea J.; Golebiowski, Diane; Izzo, Cassandra M.; Horn, Erin; Taghian, Toloo; Rodriguez, Paola; Batista, Ana Rita; Ellis, Lauren E.; Hwang, Misako; Martin, Douglas R.; Gray-Edwards, Heather; Sena-Esteves, Miguel Share Save A novel mouse model of Duchenne muscular dystrophy carrying a multi-exonic Dmd deletion exhibits progressive muscular dystrophy and early-onset cardiomyopathyWong, Tatianna Wai Ying; Ahmed, Abdalla; Yang, Grace; Maino, Eleonora; Steiman, Sydney; Hyatt, Elzbieta; Chan, Parry; Lindsay, Kyle; Wong, Nicole; Golebiowski, Diane; Schneider, Joel; Delgado-Olguin, Paul; Ivakine, Evgueni A.; Cohn, Ronald D. Share Save Share Save Share Save Optimization of AAV Vector Design for Safe Expression of β-N-Acetylhexosaminidase in the Brain for Tay-Sachs Disease Gene TherapyGolebiowski, Diane; Petrosky, Keiko; Van der Marel, Kajo; Hutto, Elizabeth; Curran, Elizabeth; Balkanska-Sinclair, Elena; Bishop, Nina; Santos, Rosemary; Macri, Sheila Cummings; Martin, Douglas; Gounis, Matthew; Pilitsis, Julie; Asaad, Wael; Sena-Esteves, Miguel Share Save 
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