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A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system.
Alterman JF, Godinho BMDC, Hassler MR, Ferguson CM, Echeverria D, Sapp E, Haraszti RA, Coles AH, Conroy F, Miller R, Roux L, Yan P, Knox EG, Turanov AA, King RM, Gernoux G, Mueller C, Gray-Edwards HL, Moser RP, Bishop NC, Jaber SM, Gounis MJ, Sena-Esteves M, Pai AA, DiFiglia M, Aronin N, Khvorova A. Alterman JF, et al. Among authors: mueller c. Nat Biotechnol. 2019 Aug;37(8):884-894. doi: 10.1038/s41587-019-0205-0. Epub 2019 Aug 2. Nat Biotechnol. 2019. PMID: 31375812 Free PMC article.
Design of shRNA and miRNA for Delivery to the CNS.
Toro Cabrera G, Mueller C. Toro Cabrera G, et al. Among authors: mueller c. Methods Mol Biol. 2016;1382:67-80. doi: 10.1007/978-1-4939-3271-9_5. Methods Mol Biol. 2016. PMID: 26611579
Regulatory and Exhausted T Cell Responses to AAV Capsid.
Gernoux G, Wilson JM, Mueller C. Gernoux G, et al. Among authors: mueller c. Hum Gene Ther. 2017 Apr;28(4):338-349. doi: 10.1089/hum.2017.022. Hum Gene Ther. 2017. PMID: 28323492 Free PMC article.
2,910 results