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Page 1
Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the mdx Mouse Model of Duchenne Muscular Dystrophy.
Hum Gene Ther. 2021 Apr;32(7-8):375-389. doi: 10.1089/hum.2019.255. Epub 2021 Feb 18.
Hum Gene Ther. 2021.
PMID: 33397205
Free PMC article.
Clinical Trial.
Preclinical Systemic Delivery of Adeno-Associated α-Sarcoglycan Gene Transfer for Limb-Girdle Muscular Dystrophy.
Griffin DA, Pozsgai ER, Heller KN, Potter RA, Peterson EL, Rodino-Klapac LR.
Griffin DA, et al. Among authors: peterson el.
Hum Gene Ther. 2021 Apr;32(7-8):390-404. doi: 10.1089/hum.2019.199. Epub 2021 Feb 18.
Hum Gene Ther. 2021.
PMID: 33349138
Free PMC article.
Clinical Trial.
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Use of plasmapheresis to lower anti-AAV antibodies in nonhuman primates with pre-existing immunity to AAVrh74.
Potter RA, Peterson EL, Griffin D, Cooper Olson G, Lewis S, Cochran K, Mendell JR, Rodino-Klapac LR.
Potter RA, et al. Among authors: peterson el.
Mol Ther Methods Clin Dev. 2024 Jan 23;32(1):101195. doi: 10.1016/j.omtm.2024.101195. eCollection 2024 Mar 14.
Mol Ther Methods Clin Dev. 2024.
PMID: 38327805
Free PMC article.
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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.
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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.
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