E. Peterson

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

J. Mendell, L. Rodino‐Klapac, R. Potter, 2017, Human gene therapy.

Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion.

J. Mendell, J. Cheatham, L. Rodino‐Klapac, 2019, Human gene therapy.

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

L. Rodino‐Klapac, A. Kempton, R. Potter, 2023, Molecular therapy. Methods & clinical development.

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

L. Rodino‐Klapac, R. Potter, E. Pozsgai, 2020, Human gene therapy.

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

J. Mendell, L. Rodino‐Klapac, R. Potter, 2021, Human gene therapy.

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

K. Flanigan, J. Cheatham, L. Rodino‐Klapac, 2018, Molecular therapy. Methods & clinical development.

Use of plasmapheresis to lower anti-AAV antibodies in nonhuman primates with pre-existing immunity to AAVrh74

E. Peterson, L. Rodino-Klapac, J. R. Mendell, 2024, Molecular therapy. Methods & clinical development.