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A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna.
Taghian T, Marosfoi MG, Puri AS, Cataltepe OI, King RM, Diffie EB, Maguire AS, Martin DR, Fernau D, Batista AR, Kuchel T, Christou C, Perumal R, Chandra S, Gamlin PD, Bertrand SG, Flotte TR, McKenna-Yasek D, Tai PWL, Aronin N, Gounis MJ, Sena-Esteves M, Gray-Edwards HL. Taghian T, et al. Among authors: aronin n. Mol Ther. 2020 Feb 5;28(2):411-421. doi: 10.1016/j.ymthe.2019.11.012. Epub 2019 Nov 16. Mol Ther. 2020. PMID: 31813800 Free PMC article.
Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits.
DiFiglia M, Sena-Esteves M, Chase K, Sapp E, Pfister E, Sass M, Yoder J, Reeves P, Pandey RK, Rajeev KG, Manoharan M, Sah DW, Zamore PD, Aronin N. DiFiglia M, et al. Among authors: aronin n. Proc Natl Acad Sci U S A. 2007 Oct 23;104(43):17204-9. doi: 10.1073/pnas.0708285104. Epub 2007 Oct 16. Proc Natl Acad Sci U S A. 2007. PMID: 17940007 Free PMC article.
Widespread Central Nervous System Gene Transfer and Silencing After Systemic Delivery of Novel AAV-AS Vector.
Choudhury SR, Harris AF, Cabral DJ, Keeler AM, Sapp E, Ferreira JS, Gray-Edwards HL, Johnson JA, Johnson AK, Su Q, Stoica L, DiFiglia M, Aronin N, Martin DR, Gao G, Sena-Esteves M. Choudhury SR, et al. Among authors: aronin n. Mol Ther. 2016 Apr;24(4):726-35. doi: 10.1038/mt.2015.231. Epub 2015 Dec 28. Mol Ther. 2016. PMID: 26708003 Free PMC article.
Effects of Exogenous NUB1 Expression in the Striatum of HDQ175/Q7 Mice.
Vodicka P, Chase K, Iuliano M, Valentine DT, Sapp E, Lu B, Kegel-Gleason KB, Sena-Esteves M, Aronin N, DiFiglia M. Vodicka P, et al. Among authors: aronin n. J Huntingtons Dis. 2016 Jun 13;5(2):163-74. doi: 10.3233/JHD-160195. J Huntingtons Dis. 2016. PMID: 27314618
Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA Silencing.
Didiot MC, Hall LM, Coles AH, Haraszti RA, Godinho BM, Chase K, Sapp E, Ly S, Alterman JF, Hassler MR, Echeverria D, Raj L, Morrissey DV, DiFiglia M, Aronin N, Khvorova A. Didiot MC, et al. Among authors: aronin n. Mol Ther. 2016 Oct;24(10):1836-1847. doi: 10.1038/mt.2016.126. Epub 2016 Jun 27. Mol Ther. 2016. PMID: 27506293 Free PMC article.
Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease.
Pfister EL, DiNardo N, Mondo E, Borel F, Conroy F, Fraser C, Gernoux G, Han X, Hu D, Johnson E, Kennington L, Liu P, Reid SJ, Sapp E, Vodicka P, Kuchel T, Morton AJ, Howland D, Moser R, Sena-Esteves M, Gao G, Mueller C, DiFiglia M, Aronin N. Pfister EL, et al. Among authors: aronin n. Hum Gene Ther. 2018 Jun;29(6):663-673. doi: 10.1089/hum.2017.199. Epub 2018 Feb 23. Hum Gene Ther. 2018. PMID: 29207890 Free PMC article.
176 results