Channel:  RNA Therapeutics Institute at UMass Chan Med School

137 Articles

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Antisense oligonucleotide targeting pathogenic sense repeat RNA in C9ORF72 suppresses production of antisense-dependent dipeptide repeat proteins implicated in ALS/FTD
Gu, Y., Kankel, M. W., Watts, J., Jafar-nejad, P., Almeida, S.
10.1101/2024.10.04.616663 — Posted: 2024-10-05
Next generation APOBEC3 inhibitors: Optimally designed for potency and nuclease stability
Hedger, A. K., Myint, W., Lee, J. M., Suchenski-Loustaunau, D., Balachandran, V., Shaqra, A. M., Kurt Yilmaz, N., Watts, J., Matsuo, H., Schiffer, C. A.
10.1101/2024.09.05.611238 — Posted: 2024-09-06
Amortized template-matching of molecular conformations from cryo-electron microscopy images using simulation-based inference
Dingeldein, L., Silva-Sanchez, D., Evans, L., D'Imprima, E., Grigorieff, N., Covino, R., Cossio, P.
10.1101/2024.07.23.604154 — Posted: 2024-07-31
PRC1.6 localizes on chromatin with the human silencing hub (HUSH) complex for promoter-specific silencing
Rodriguez, T. C., Yurkovetskiy, L., Nagalekshmi, K., Lam, C. H. O., Jazbec, E., Maitland, S. A., Wolfe, S., Sontheimer, E. J., Luban, J.
10.1101/2024.07.12.603173 — Posted: 2024-07-13
RNAi-mediated silencing of SOD1 profoundly extends survival and functional outcomes in ALS mice
Weiss, A., Gilbert, J. W., Flores, I. V. R., Belgrad, J., Ferguson, C., Dogan, E. O., Wightman, N., Mocarski, K., Echeverria, D., Summers, A., Bramato, B., McHugh, N., Furgal, R., Yamada, N., Cooper, D., Monopoli, K., Godinho, B. M. D. C., Hassler, M. R., Yamada, K., Greer, P. L., Henninger, N., Brown, R. H., Khvorova, A.
10.1101/2024.06.20.599943 — Posted: 2024-06-25
Preventing acute neurotoxicity of CNS therapeutic oligonucleotides with the addition of Ca2+ and Mg2+ in the formulation
Miller, R., Paquette, J., Barker, A., Sapp, E., McHugh, N., Bramato, B., Yamada, N., Alterman, J., Echeveria, D., Yamada, K., Watts, J. K., Anaclet, C., DiFiglia, M., Khvorova, A., Aronin, N.
10.1101/2024.06.06.597639 — Posted: 2024-06-08
mRNA nuclear clustering leads to a difference in mutant huntingtin mRNA and protein silencing by siRNAs in vivo
Allen, S. J., O'Reilly, D., Miller, R., Sapp, E., Summers, A., Paquette, J., Moreno, D. E., Bramato, B., McHugh, N., Yamada, K., Aronin, N., DiFiglia, M., Khvorova, A.
10.1101/2024.04.24.590997 — Posted: 2024-04-28
Cryo-EM Structure of HRSL Domain Reveals Activating Crossed Helices at the Core of GCN2
Solorio - Kirpichyan, K., Golovenko, D., Korostelev, A., Yan, N., Korennykh, A.
10.1101/2024.04.24.591037 — Posted: 2024-04-25
Traumatic injury causes selective degeneration and TDP-43 mislocalization in human iPSC-derived C9orf72-associated ALS/FTD motor neurons
Martin, E. J., Santacruz, C., Mitevska, A., Jones, I. E., Krishnan, G., Gao, F.-B., Finan, J. D., Kiskinis, E.
10.1101/2024.03.21.586073 — Posted: 2024-03-26
mRNA initiation and termination are spatially coordinated
Calvo-Roitberg, E., Carroll, C. L., Venev, S. V., Kim, G., Mick, S. T., Dekker, J., Fiszbein, A., Pai, A. A.
10.1101/2024.01.05.574404 — Posted: 2024-01-07

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