Below are the most recent publications written about "Huntingtin Protein" by people in Profiles.
-
Papadopoulou AS, Alterman J, Landles C, Smith EJ, Conroy F, Phillips J, Canibano-Pico M, Nita IM, Osborne GF, Iqbal A, Aldous SG, Bondulich MK, Gomez-Paredes C, Sathasivam K, O'Reilly D, Echeverria D, Bobkov K, Greene JR, Aronin N, Khvorova A, Bates GP. Lowering the HTT1a transcript as an effective therapy for Huntington's disease in a knockin mouse model. Sci Transl Med. 2026 Mar 18; 18(841):eadw2495.
-
Mathews EW, Coffey SR, G?rtner A, Belgrad J, Bragg RM, O'Reilly D, Cantle JP, McHugh C, Summers A, Fentz J, Schwagarus T, Cornelius A, Lingos I, Burch Z, Kovalenko M, Andrew MA, Bennett CF, Kordasiewicz HB, Marchionini DM, Wilkinson H, Vogt TF, Beuzer P, Pinto RM, Khvorova A, Howland D, Wheeler VC, Carroll JB. Suppression of Huntington's Disease Somatic Instability by Transcriptional Repression and Direct CAG Repeat Binding. Nat Commun. 2025 Nov 14; 16(1):10009.
-
Iwanowicz A, Boudi A, Seeley C, Sapp E, Miller R, Liu S, Chase K, Shing K, Batista AR, Siena-Esteves M, Aronin N, DiFiglia M, Kegel-Gleason KB. Intrastriatal Delivery of a Zinc Finger Protein Targeting the Mutant HTT Gene Allele Obviates Lipid Phenotypes in Brain and Plasma in Huntington's Disease?Mice. Hum Gene Ther. 2025 08; 36(15-16):1083-1094.
-
Allen S, O'Reilly D, Miller R, Sapp E, Summers A, Paquette J, Echeverria Moreno D, Bramato B, McHugh N, Yamada K, Aronin N, DiFiglia M, Khvorova A. mRNA Nuclear Clustering Leads to a Difference in Mutant Huntingtin mRNA and Protein Silencing by siRNAs In Vivo. Nucleic Acid Ther. 2024 08; 34(4):164-172.
-
Belgrad J, Tang Q, Hildebrand S, Summers A, Sapp E, Echeverria D, O'Reilly D, Luu E, Bramato B, Allen S, Cooper D, Alterman J, Yamada K, Aronin N, DiFiglia M, Khvorova A. A programmable dual-targeting siRNA scaffold supports potent two-gene modulation in the central nervous system. Nucleic Acids Res. 2024 06 24; 52(11):6099-6113.
-
Belgrad J, Khvorova A. More than 185 CAG repeats: a point of no return in Huntington's disease biology. Brain. 2024 05 03; 147(5):1601-1603.
-
O'Reilly D, Belgrad J, Ferguson C, Summers A, Sapp E, McHugh C, Mathews E, Boudi A, Buchwald J, Ly S, Moreno D, Furgal R, Luu E, Kennedy Z, Hariharan V, Monopoli K, Yang XW, Carroll J, DiFiglia M, Aronin N, Khvorova A. Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington's disease. Mol Ther. 2023 06 07; 31(6):1661-1674.
-
Sapp E, Boudi A, Reid SJ, Trombetta BA, Kivis?kk P, Taghian T, Arnold SE, Howland D, Gray-Edwards H, Kegel-Gleason KB, DiFiglia M. Levels of Synaptic Proteins in Brain and Neurofilament Light Chain in Cerebrospinal Fluid and Plasma of OVT73 Huntington's Disease Sheep Support a Prodromal Disease State. J Huntingtons Dis. 2023; 12(3):201-213.
-
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. Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington's disease models. Nat Commun. 2022 10 03; 13(1):5802.
-
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. Allele-Specific Knockdown of Mutant Huntingtin Protein via Editing at Coding Region Single Nucleotide Polymorphism Heterozygosities. Hum Gene Ther. 2022 01; 33(1-2):25-36.