Detalhe da pesquisa
1.
An RNAi therapeutic targeting hepatic DGAT2 in a genetically obese mouse model of nonalcoholic steatohepatitis.
Mol Ther
; 30(3): 1329-1342, 2022 03 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-34774753
2.
Structurally constrained phosphonate internucleotide linkage impacts oligonucleotide-enzyme interaction, and modulates siRNA activity and allele specificity.
Nucleic Acids Res
; 49(21): 12069-12088, 2021 12 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-34850120
3.
Loss of huntingtin function slows synaptic vesicle endocytosis in striatal neurons from the httQ140/Q140 mouse model of Huntington's disease.
Neurobiol Dis
; 134: 104637, 2020 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-31614197
4.
Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo.
Nucleic Acids Res
; 46(5): 2185-2196, 2018 03 16.
Artigo
em Inglês
| MEDLINE | ID: mdl-29432571
5.
Optimized Cholesterol-siRNA Chemistry Improves Productive Loading onto Extracellular Vesicles.
Mol Ther
; 26(8): 1973-1982, 2018 08 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-29937418
6.
Transvascular Delivery of Hydrophobically Modified siRNAs: Gene Silencing in the Rat Brain upon Disruption of the Blood-Brain Barrier.
Mol Ther
; 26(11): 2580-2591, 2018 11 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-30143435
7.
5Î-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo.
Nucleic Acids Res
; 45(13): 7581-7592, 2017 Jul 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-28591791
8.
Visualization of self-delivering hydrophobically modified siRNA cellular internalization.
Nucleic Acids Res
; 45(1): 15-25, 2017 01 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-27899655
9.
Exosome-mediated Delivery of Hydrophobically Modified siRNA for Huntingtin mRNA Silencing.
Mol Ther
; 24(10): 1836-1847, 2016 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-27506293
10.
Guanabenz (Wytensin™) selectively enhances uptake and efficacy of hydrophobically modified siRNAs.
Nucleic Acids Res
; 43(18): 8664-72, 2015 Oct 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-26400165
11.
Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in mouse and pig photoreceptors.
Mol Ther Nucleic Acids
; 35(1): 102088, 2024 Mar 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-38192611
12.
Multispecies-targeting siRNAs for the modulation of JAK1 in the skin.
Mol Ther Nucleic Acids
; 35(1): 102117, 2024 Mar 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-38304729
13.
Single intravitreal administration of a tetravalent siRNA exhibits robust and efficient gene silencing in rodent and swine photoreceptors.
bioRxiv
; 2023 Sep 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-37790464
14.
Extended Nucleic Acid (exNA): A Novel, Biologically Compatible Backbone that Significantly Enhances Oligonucleotide Efficacy in vivo.
bioRxiv
; 2023 May 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-37292886
15.
Rational design of a JAK1-selective siRNA inhibitor for the modulation of autoimmunity in the skin.
Nat Commun
; 14(1): 7099, 2023 11 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-37925520
16.
Chemical optimization of siRNA for safe and efficient silencing of placental sFLT1.
Mol Ther Nucleic Acids
; 29: 135-149, 2022 Sep 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-35847173
17.
PK-modifying anchors significantly alter clearance kinetics, tissue distribution, and efficacy of therapeutics siRNAs.
Mol Ther Nucleic Acids
; 29: 116-132, 2022 Sep 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-35795486
18.
Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington's disease models.
Nat Commun
; 13(1): 5802, 2022 10 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-36192390
19.
Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep.
JCI Insight
; 6(24)2021 12 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-34935646
20.
2'-O-Methyl at 20-mer Guide Strand 3' Termini May Negatively Affect Target Silencing Activity of Fully Chemically Modified siRNA.
Mol Ther Nucleic Acids
; 21: 266-277, 2020 Sep 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-32610253