MicroRNA Based Treatment of Cardiomyopathy: Not all Dystrophies are Created Equal

por Rodino‐Klapac, Louise R.
Publicado 2013

Evaluation of the Lipid-binding Properties of Recombinant Dystrophin Spectrin-like Repeat Domains R1-3

por Cooper-Olson, Grace, Rodino-Klapac, Louise R., Potter, Rachael A.
Publicado 2021

Follistatin Gene Therapy Improves Ambulation in Becker Muscular Dystrophy

por Al-Zaidy, Samiah A., Sahenk, Zarife, Rodino-Klapac, Louise R., Kaspar, Brian, Mendell, Jerry R.
Publicado 2015

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

Assessment of rAAVrh.74.MHCK7.micro-dystrophin Gene Therapy Using Magnetic Resonance Imaging in Children With Duchenne Muscular Dystrophy

por Willcocks, Rebecca J., Forbes, Sean C., Walter, Glenn A., Sweeney, Lee, Rodino-Klapac, Louise R., Mendell, Jerry R., Vandenborne, Krista
Publicado 2021

A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy

por Rodino-Klapac, Louise R, Janssen, Paul ML, Montgomery, Chrystal L, Coley, Brian D, Chicoine, Louis G, Clark, K Reed, Mendell, Jerry R
Publicado 2007

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

Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States

por Klimchak, Alexa C., Sedita, Lauren E., Rodino-Klapac, Louise R., Mendell, Jerry R., McDonald, Craig M., Gooch, Katherine L., Malone, Daniel C.
Publicado 2023

AAV1.NT-3 Gene Therapy for Charcot–Marie–Tooth Neuropathy

por Sahenk, Zarife, Galloway, Gloria, Clark, Kelly Reed, Malik, Vinod, Rodino-Klapac, Louise R, Kaspar, Brian K., Chen, Lei, Braganza, Cilwyn, Montgomery, Chrystal, Mendell, Jerry R
Publicado 2014

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

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

Persistent Expression of FLAG-tagged Micro dystrophin in Nonhuman Primates Following Intramuscular and Vascular Delivery

por Rodino-Klapac, Louise R, Montgomery, Chrystal L, Bremer, William G, Shontz, Kimberly M, Malik, Vinod, Davis, Nancy, Sprinkle, Spencer, Campbell, Katherine J, Sahenk, Zarife, Clark, K Reed, Walker, Christopher M, Mendell, Jerry R, Chicoine, Louis G
Publicado 2009

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

Current Clinical Applications of In Vivo Gene Therapy with AAVs

por Mendell, Jerry R., Al-Zaidy, Samiah A., Rodino-Klapac, Louise R., Goodspeed, Kimberly, Gray, Steven J., Kay, Christine N., Boye, Sanford L., Boye, Shannon E., George, Lindsey A., Salabarria, Stephanie, Corti, Manuela, Byrne, Barry J., Tremblay, Jacques P.
Publicado 2021

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