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Allele-Specific Knockdown of Mutant Huntingtin Protein via Editing at Coding Region Single Nucleotide Polymorphism Heterozygosities.
Oikemus SR, Pfister EL, Sapp E, Chase KO, Kennington LA, Hudgens E, Miller R, Zhu LJ, Chaudhary A, Mick EO, Sena-Esteves M, Wolfe SA, DiFiglia M, Aronin N, Brodsky MH. Oikemus SR, et al. Among authors: pfister el. Hum Gene Ther. 2022 Jan;33(1-2):25-36. doi: 10.1089/hum.2020.323. Hum Gene Ther. 2022. PMID: 34376056 Free PMC article.
Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington's disease models.
Conroy F, Miller R, Alterman JF, Hassler MR, Echeverria D, Godinho BMDC, Knox EG, Sapp E, Sousa J, Yamada K, Mahmood F, Boudi A, Kegel-Gleason K, DiFiglia M, Aronin N, Khvorova A, Pfister EL. Conroy F, et al. Among authors: pfister el. Nat Commun. 2022 Oct 3;13(1):5802. doi: 10.1038/s41467-022-33061-x. Nat Commun. 2022. PMID: 36192390 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: pfister e. 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.
Huntington's Disease: Les Jeux Sont Faits?
Pfister EL, Aronin N. Pfister EL, et al. Trends Mol Med. 2020 Oct;26(10):889-890. doi: 10.1016/j.molmed.2020.08.004. Epub 2020 Sep 4. Trends Mol Med. 2020. PMID: 32893090
147 results