Expression and function of four AAV-based constructs for dystrophin restoration in the mdx mouse model of Duchenne muscular dystrophy

por Potter, Rachael A., Griffin, Danielle A., Heller, Kristin N., Mendell, Jerry R., Rodino-Klapac, Louise R.
Publicado 2023

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

Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the mdx Mouse Model of Duchenne Muscular Dystrophy

por Potter, Rachael A., Griffin, Danielle A., Heller, Kristin N., Peterson, Ellyn L., Clark, Emma K., Mendell, Jerry R., Rodino-Klapac, Louise R.
Publicado 2021

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

A novel DMD IRES results in a functional N-truncated dystrophin, providing a potential route to therapy for patients with 5’ mutations

por Wein, Nicolas, Vulin, Adeline, Sofia Falzarano, Maria, Al-Khalili Szigyarto, Christina, Maiti, Baijayanta, Findlay, Andrew, Heller, Kristin N, Uhlén, Mathias, Bakthavachalu, Baskar, Messina, Sonia, Vita, Giuseppe, Passarelli, Chiara, Gualandi, Francesca, Wilton, Steve D, Rodino-Klapac, Louise, Yang, Lin, Dunn, Diane M., Schoenberg, Daniel, Weiss, Robert B., Howard, Michael T., Ferlini, Alessandra, Flanigan, Kevin M.
Publicado 2014