Preclinical Systemic Delivery of Adeno-Associated α-Sarcoglycan Gene Transfer for Limb-Girdle Muscular Dystrophy

por Griffin, Danielle A., Pozsgai, Eric R., Heller, Kristin N., Potter, Rachael A., Peterson, Ellyn L., Rodino-Klapac, Louise R.
Publicado 2021

Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse model

por Seo, Young-Eun, Baine, Stephen H., Kempton, Amber N., Rogers, Oliver C., Lewis, Sarah, Adegboye, Kaitlin, Haile, Alex, Griffin, Danielle A., Peterson, Ellyn L., Pozsgai, Eric R., Potter, Rachael A., Rodino-Klapac, Louise R.
Publicado 2023

AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models

por Sondergaard, Patricia C, Griffin, Danielle A, Pozsgai, Eric R, Johnson, Ryan W, Grose, William E, Heller, Kristin N, Shontz, Kim M, Montgomery, Chrystal L, Liu, Joseph, Clark, Kelly Reed, Sahenk, Zarife, Mendell, Jerry R, Rodino-Klapac, Louise R
Publicado 2015

Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Gene Expression and Functional Improvement for Dysferlinopathy

por Potter, Rachael A., Griffin, Danielle A., Sondergaard, Patricia C., Johnson, Ryan W., Pozsgai, Eric R., Heller, Kristin N., Peterson, Ellyn L., Lehtimäki, Kimmo K., Windish, Hillarie P., Mittal, Plavi J., Albrecht, Douglas E., Mendell, Jerry R., Rodino-Klapac, Louise R.
Publicado 2018

Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial

por Mendell, Jerry R., Sahenk, Zarife, Lehman, Kelly, Nease, Carrie, Lowes, Linda P., Miller, Natalie F., Iammarino, Megan A., Alfano, Lindsay N., Nicholl, Amanda, Al-Zaidy, Samiah, Lewis, Sarah, Church, Kathleen, Shell, Richard, Cripe, Linda H., Potter, Rachael A., Griffin, Danielle A., Pozsgai, Eric, Dugar, Ashish, Hogan, Mark, Rodino-Klapac, Louise R.
Publicado 2020