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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. Among authors: rinaldi f. 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.
Excess α-synuclein compromises phagocytosis in iPSC-derived macrophages.
Haenseler W, Zambon F, Lee H, Vowles J, Rinaldi F, Duggal G, Houlden H, Gwinn K, Wray S, Luk KC, Wade-Martins R, James WS, Cowley SA. Haenseler W, et al. Among authors: rinaldi f. Sci Rep. 2017 Aug 21;7(1):9003. doi: 10.1038/s41598-017-09362-3. Sci Rep. 2017. PMID: 28827786 Free PMC article.
High content analysis in amyotrophic lateral sclerosis.
Rinaldi F, Motti D, Ferraiuolo L, Kaspar BK. Rinaldi F, et al. Mol Cell Neurosci. 2017 Apr;80:180-191. doi: 10.1016/j.mcn.2016.12.001. Epub 2016 Dec 11. Mol Cell Neurosci. 2017. PMID: 27965018 Free PMC article. Review.
A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.GALGT2.
Flanigan KM, Vetter TA, Simmons TR, Iammarino M, Frair EC, Rinaldi F, Chicoine LG, Harris J, Cheatham JP, Cheatham SL, Boe B, Waldrop MA, Zygmunt DA, Packer D, Martin PT. Flanigan KM, et al. Among authors: rinaldi f. Mol Ther Methods Clin Dev. 2022 Sep 2;27:47-60. doi: 10.1016/j.omtm.2022.08.009. eCollection 2022 Dec 8. Mol Ther Methods Clin Dev. 2022. PMID: 36186954 Free PMC article.
523 results