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Page 1
Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN.
Foust KD, Wang X, McGovern VL, Braun L, Bevan AK, Haidet AM, Le TT, Morales PR, Rich MM, Burghes AH, Kaspar BK. Foust KD, et al. Among authors: morales pr. Nat Biotechnol. 2010 Mar;28(3):271-4. doi: 10.1038/nbt.1610. Epub 2010 Feb 28. Nat Biotechnol. 2010. Retraction in: Nat Biotechnol. 2022 Nov;40(11):1692. doi: 10.1038/s41587-022-01497-7 PMID: 20190738 Free PMC article. Retracted.
Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease.
Cain JT, Likhite S, White KA, Timm DJ, Davis SS, Johnson TB, Dennys-Rivers CN, Rinaldi F, Motti D, Corcoran S, Morales P, Pierson C, Hughes SM, Lee SY, Kaspar BK, Meyer K, Weimer JM. Cain JT, et al. Mol Ther. 2019 Oct 2;27(10):1836-1847. doi: 10.1016/j.ymthe.2019.06.015. Epub 2019 Jul 10. Mol Ther. 2019. PMID: 31331814 Free PMC article.
Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates.
Powers S, Likhite S, Gadalla KK, Miranda CJ, Huffenberger AJ, Dennys C, Foust KD, Morales P, Pierson CR, Rinaldi F, Perry S, Bolon B, Wein N, Cobb S, Kaspar BK, Meyer KC. Powers S, et al. Mol Ther. 2023 Sep 6;31(9):2767-2782. doi: 10.1016/j.ymthe.2023.07.013. Epub 2023 Jul 22. Mol Ther. 2023. PMID: 37481701 Free PMC article.
Clade F AAVHSCs cross the blood brain barrier and transduce the central nervous system in addition to peripheral tissues following intravenous administration in nonhuman primates.
Ellsworth JL, Gingras J, Smith LJ, Rubin H, Seabrook TA, Patel K, Zapata N, Olivieri K, O'Callaghan M, Chlipala E, Morales P, Seymour A. Ellsworth JL, et al. PLoS One. 2019 Nov 26;14(11):e0225582. doi: 10.1371/journal.pone.0225582. eCollection 2019. PLoS One. 2019. PMID: 31770409 Free PMC article.
Assessment of a venous thromboembolism prophylaxis shared decision-making intervention (DASH-TOP) using the decisional conflict scale: a mixed-method study.
León-García M, Humphries B, Morales PR, Gravholt D, Eckman MH, Bates SM, Suárez NRE, Xie F, Perestelo-Pérez L, Alonso-Coello P. León-García M, et al. Among authors: morales pr. BMC Med Inform Decis Mak. 2023 Nov 6;23(1):250. doi: 10.1186/s12911-023-02349-3. BMC Med Inform Decis Mak. 2023. PMID: 37932759 Free PMC article. Clinical Trial.
18 results