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Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR).
Zaidman CM, Proud CM, McDonald CM, Lehman KJ, Goedeker NL, Mason S, Murphy AP, Guridi M, Wang S, Reid C, Darton E, Wandel C, Lewis S, Malhotra J, Griffin DA, Potter RA, Rodino-Klapac LR, Mendell JR. Zaidman CM, et al. Among authors: griffin da. Ann Neurol. 2023 Nov;94(5):955-968. doi: 10.1002/ana.26755. Epub 2023 Sep 7. Ann Neurol. 2023. PMID: 37539981
Vascular delivery of rAAVrh74.MCK.GALGT2 to the gastrocnemius muscle of the rhesus macaque stimulates the expression of dystrophin and laminin α2 surrogates.
Chicoine LG, Rodino-Klapac LR, Shao G, Xu R, Bremer WG, Camboni M, Golden B, Montgomery CL, Shontz K, Heller KN, Griffin DA, Lewis S, Coley BD, Walker CM, Clark KR, Sahenk Z, Mendell JR, Martin PT. Chicoine LG, et al. Among authors: griffin da. Mol Ther. 2014 Apr;22(4):713-24. doi: 10.1038/mt.2013.246. Epub 2013 Oct 22. Mol Ther. 2014. PMID: 24145553 Free PMC article.
Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular delivery.
Chicoine LG, Montgomery CL, Bremer WG, Shontz KM, Griffin DA, Heller KN, Lewis S, Malik V, Grose WE, Shilling CJ, Campbell KJ, Preston TJ, Coley BD, Martin PT, Walker CM, Clark KR, Sahenk Z, Mendell JR, Rodino-Klapac LR. Chicoine LG, et al. Among authors: griffin da. Mol Ther. 2014 Feb;22(2):338-347. doi: 10.1038/mt.2013.244. Epub 2013 Oct 23. Mol Ther. 2014. PMID: 24196577 Free PMC article.
AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models.
Sondergaard PC, Griffin DA, Pozsgai ER, Johnson RW, Grose WE, Heller KN, Shontz KM, Montgomery CL, Liu J, Clark KR, Sahenk Z, Mendell JR, Rodino-Klapac LR. Sondergaard PC, et al. Among authors: griffin da. Ann Clin Transl Neurol. 2015 Mar;2(3):256-70. doi: 10.1002/acn3.172. Epub 2015 Jan 20. Ann Clin Transl Neurol. 2015. PMID: 25815352 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: griffin da. 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.
45 results