Rational design of a JAK1-selective siRNA inhibitor for the modulation of autoimmunity in the skin.

Autor: Tang Q; Department of Dermatology, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA.; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Fakih HH; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Zain Ui Abideen M; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Hildebrand SR; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Afshari K; Department of Dermatology, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Gross KY; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Sousa J; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Maebius AS; Program in Molecular Medicine, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Bartholdy C; LEO Pharma A/S, Industriparken 55, 2750, Ballerup, Denmark., Søgaard PP; LEO Pharma A/S, Industriparken 55, 2750, Ballerup, Denmark., Jackerott M; LEO Pharma A/S, Industriparken 55, 2750, Ballerup, Denmark., Hariharan V; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Summers A; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Fan X; Department of Dermatology, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Okamura K; Department of Dermatology, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Monopoli KR; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA.; Bioinformatics and Computational Biology Program, Worcester Polytechnic Institute, Worcester, MA, 01609, USA., Cooper DA; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA.; Program in Molecular Medicine, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Echeverria D; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Bramato B; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., McHugh N; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Furgal RC; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Dresser K; Department of Pathology, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Winter SJ; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Biscans A; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Chuprin J; Department of Dermatology, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Haddadi NS; Department of Dermatology, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Sherman S; Department of Dermatology, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Yıldız-Altay Ü; Department of Dermatology, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Rashighi M; Department of Dermatology, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Richmond JM; Department of Dermatology, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA., Bouix-Peter C; Aldena Therapeutics, London, E1 6RA, UK., Blanchard C; Aldena Therapeutics, London, E1 6RA, UK., Clauss A; LEO Pharma A/S, Industriparken 55, 2750, Ballerup, Denmark., Alterman JF; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA. Julia.Alterman@umassmed.edu., Khvorova A; RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA. Anastasia.Khvorova@umassmed.edu.; Program in Molecular Medicine, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA. Anastasia.Khvorova@umassmed.edu., Harris JE; Department of Dermatology, University of Massachusetts Chan Medical School, Worcester, MA, 01605, USA. John.Harris@umassmed.edu.

Publikováno v: Nature communications [Nat Commun] 2023 Nov 04; Vol. 14 (1), pp. 7099. Date of Electronic Publication: 2023 Nov 04.

mRNA Nuclear Clustering Leads to a Difference in Mutant Huntingtin mRNA and Protein Silencing by siRNAs In Vivo.

Autor: Allen, Sarah, O'Reilly, Daniel, Miller, Rachael, Sapp, Ellen, Summers, Ashley, Paquette, Joseph, Echeverria Moreno, Dimas, Bramato, Brianna, McHugh, Nicholas, Yamada, Ken, Aronin, Neil, DiFiglia, Marian, Khvorova, Anastasia

Publikováno v: Nucleic Acid Therapeutics; Aug2024, Vol. 34 Issue 4, p164-172, 9p

Quantifying the activity profile of ASO and siRNA conjugates in glioblastoma xenograft tumors in vivo.

Autor: Sarli, Samantha L, Fakih, Hassan H, Kelly, Karen, Devi, Gitali, Rembetsy-Brown, Julia M, McEachern, Holly R, Ferguson, Chantal M, Echeverria, Dimas, Lee, Jonathan, Sousa, Jacquelyn, Sleiman, Hanadi F, Khvorova, Anastasia, Watts, Jonathan K

Publikováno v: Nucleic Acids Research; 5/22/2024, Vol. 52 Issue 9, p4799-4817, 19p