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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.
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.
An Isolated Limb Infusion Method Allows for Broad Distribution of rAAVrh74.MCK.GALGT2 to Leg Skeletal Muscles in the Rhesus Macaque.
Xu R, Jia Y, Zygmunt DA, Cramer ML, Crowe KE, Shao G, Maki AE, Guggenheim HN, Hood BC, Griffin DA, Peterson E, Bolon B, Cheatham JP, Cheatham SL, Flanigan KM, Rodino-Klapac LR, Chicoine LG, Martin PT. Xu R, et al. Mol Ther Methods Clin Dev. 2018 Jul 14;10:89-104. doi: 10.1016/j.omtm.2018.06.002. eCollection 2018 Sep 21. Mol Ther Methods Clin Dev. 2018. PMID: 30073180 Free PMC article.
Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion.
Mendell JR, Chicoine LG, Al-Zaidy SA, Sahenk Z, Lehman K, Lowes L, Miller N, Alfano L, Galliers B, Lewis S, Murrey D, Peterson E, Griffin DA, Church K, Cheatham S, Cheatham J, Hogan MJ, Rodino-Klapac LR. Mendell JR, et al. Hum Gene Ther. 2019 Jul;30(7):794-801. doi: 10.1089/hum.2019.006. Epub 2019 Apr 19. Hum Gene Ther. 2019. PMID: 30838895 Free PMC article.
260 results