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

Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations

por Mendell, Jerry R., Connolly, Anne M., Lehman, Kelly J., Griffin, Danielle A., Khan, Sohrab Z., Dharia, Sachi D., Quintana-Gallardo, Lucía, Rodino-Klapac, Louise R.
Publicado 2022

Evaluation of rAAVrh74 gene therapy vector seroprevalence by measurement of total binding antibodies in patients with Duchenne muscular dystrophy

por Goedeker, Natalie L., Dharia, Sachi D., Griffin, Danielle A., Coy, Jesantha, Truesdale, Todd, Parikh, Rajan, Whitehouse, Kasen, Santra, Sourav, Asher, Damon R., Zaidman, Craig M.
Publicado 2023

Homologous Recombination Mediates Functional Recovery of Dysferlin Deficiency following AAV5 Gene Transfer

por Grose, William E., Clark, K. Reed, Griffin, Danielle, Malik, Vinod, Shontz, Kimberly M., Montgomery, Chrystal L., Lewis, Sarah, Brown, Robert H., Janssen, Paul M. L., Mendell, Jerry R., Rodino-Klapac, Louise R.
Publicado 2012

Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD

por 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.
Publicado 2017

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

An Isolated Limb Infusion Method Allows for Broad Distribution of rAAVrh74.MCK.GALGT2 to Leg Skeletal Muscles in the Rhesus Macaque

por Xu, Rui, Jia, Ying, Zygmunt, Deborah A., Cramer, Megan L., Crowe, Kelly E., Shao, Guohong, Maki, Agatha E., Guggenheim, Haley N., Hood, Benjamin C., Griffin, Danielle A., Peterson, Ellyn, Bolon, Brad, Cheatham, John P., Cheatham, Sharon L., Flanigan, Kevin M., Rodino-Klapac, Louise R., Chicoine, Louis G., Martin, Paul T.
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

Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscu...

por Mendell, Jerry R., Shieh, Perry B., McDonald, Craig M., Sahenk, Zarife, Lehman, Kelly J., Lowes, Linda P., Reash, Natalie F., Iammarino, Megan A., Alfano, Lindsay N., Sabo, Brenna, Woods, Jeremy D., Skura, Christy L., Mao, Howard C., Staudt, Loretta A., Griffin, Danielle A., Lewis, Sarah, Wang, Shufang, Potter, Rachael A., Singh, Teji, Rodino-Klapac, Louise R.
Publicado 2023