Detalhe da pesquisa
1.
Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression.
Cell
; 150(5): 895-908, 2012 Aug 31.
Artigo
em Inglês
| MEDLINE | ID: mdl-22939619
2.
Single-stranded siRNAs activate RNAi in animals.
Cell
; 150(5): 883-94, 2012 Aug 31.
Artigo
em Inglês
| MEDLINE | ID: mdl-22939618
3.
Systematic Investigation of Tether Length and Phosphorus Configuration in Backbone Constrained Macrocyclic Nucleic Acids to Modulate Binding Kinetics for RNA.
J Org Chem
; 88(6): 3599-3614, 2023 03 17.
Artigo
em Inglês
| MEDLINE | ID: mdl-36857642
4.
The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration.
Nucleic Acids Res
; 49(2): 657-673, 2021 01 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-33367834
5.
Towards next generation antisense oligonucleotides: mesylphosphoramidate modification improves therapeutic index and duration of effect of gapmer antisense oligonucleotides.
Nucleic Acids Res
; 49(16): 9026-9041, 2021 09 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-34417625
6.
Site-specific incorporation of 5'-methyl DNA enhances the therapeutic profile of gapmer ASOs.
Nucleic Acids Res
; 49(4): 1828-1839, 2021 02 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-33544849
7.
Backbone Hydrocarbon-Constrained Nucleic Acids Modulate Hybridization Kinetics for RNA.
J Am Chem Soc
; 144(4): 1941-1950, 2022 02 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-35041415
8.
Targeting Huntingtin Expression in Patients with Huntington's Disease.
N Engl J Med
; 380(24): 2307-2316, 2019 06 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-31059641
9.
Mechanisms of palmitic acid-conjugated antisense oligonucleotide distribution in mice.
Nucleic Acids Res
; 48(8): 4382-4395, 2020 05 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-32182359
10.
Understanding the effect of controlling phosphorothioate chirality in the DNA gap on the potency and safety of gapmer antisense oligonucleotides.
Nucleic Acids Res
; 48(4): 1691-1700, 2020 02 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-31980820
11.
Characterization of the interactions of chemically-modified therapeutic nucleic acids with plasma proteins using a fluorescence polarization assay.
Nucleic Acids Res
; 47(3): 1110-1122, 2019 02 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-30566688
12.
Fatty acid conjugation enhances potency of antisense oligonucleotides in muscle.
Nucleic Acids Res
; 47(12): 6029-6044, 2019 07 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-31127296
13.
Site-specific replacement of phosphorothioate with alkyl phosphonate linkages enhances the therapeutic profile of gapmer ASOs by modulating interactions with cellular proteins.
Nucleic Acids Res
; 47(11): 5465-5479, 2019 06 20.
Artigo
em Inglês
| MEDLINE | ID: mdl-31034558
14.
Conjugation of hydrophobic moieties enhances potency of antisense oligonucleotides in the muscle of rodents and non-human primates.
Nucleic Acids Res
; 47(12): 6045-6058, 2019 07 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-31076766
15.
Convective forces increase rostral delivery of intrathecal radiotracers and antisense oligonucleotides in the cynomolgus monkey nervous system.
J Transl Med
; 18(1): 309, 2020 08 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-32771027
16.
A modular analysis of microglia gene expression, insights into the aged phenotype.
BMC Genomics
; 20(1): 164, 2019 Feb 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-30819113
17.
A novel humanized mouse model of Huntington disease for preclinical development of therapeutics targeting mutant huntingtin alleles.
Hum Mol Genet
; 26(6): 1115-1132, 2017 03 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-28104789
18.
Characterizing the effect of GalNAc and phosphorothioate backbone on binding of antisense oligonucleotides to the asialoglycoprotein receptor.
Nucleic Acids Res
; 45(5): 2294-2306, 2017 03 17.
Artigo
em Inglês
| MEDLINE | ID: mdl-28158620
19.
S-Acyl-2-Thioethyl: A Convenient Base-Labile Protecting Group for the Synthesis of siRNAs Containing 5'-Vinylphosphonate.
Molecules
; 24(2)2019 Jan 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-30634486
20.
Evaluation of the effect of 2'-O-methyl, fluoro hexitol, bicyclo and Morpholino nucleic acid modifications on potency of GalNAc conjugated antisense oligonucleotides in mice.
Bioorg Med Chem Lett
; 28(23-24): 3774-3779, 2018 12 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-30342955