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Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Gene Expression and Functional Improvement for Dysferlinopathy.
Potter RA, Griffin DA, Sondergaard PC, Johnson RW, Pozsgai ER, Heller KN, Peterson EL, Lehtimäki KK, Windish HP, Mittal PJ, Albrecht DE, Mendell JR, Rodino-Klapac LR. Potter RA, et al. Among authors: peterson el. Hum Gene Ther. 2018 Jul;29(7):749-762. doi: 10.1089/hum.2017.062. Epub 2017 Jul 13. Hum Gene Ther. 2018. PMID: 28707952 Free PMC article.
Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse model.
Seo YE, Baine SH, Kempton AN, Rogers OC, Lewis S, Adegboye K, Haile A, Griffin DA, Peterson EL, Pozsgai ER, Potter RA, Rodino-Klapac LR. Seo YE, et al. Among authors: peterson el. Mol Ther Methods Clin Dev. 2023 Jan 16;28:284-299. doi: 10.1016/j.omtm.2023.01.004. eCollection 2023 Mar 9. Mol Ther Methods Clin Dev. 2023. PMID: 36816759 Free PMC article.