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.
GalNAc-conjugated siRNA targeting the DNAJB1-PRKACA fusion junction in fibrolamellar hepatocellular carcinoma.
Mol Ther
; 32(1): 140-151, 2024 Jan 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-37980543
4.
Premature transcription termination at the expanded GAA repeats and aberrant alternative polyadenylation contributes to the Frataxin transcriptional deficit in Friedreich's ataxia.
Hum Mol Genet
; 31(20): 3539-3557, 2022 10 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-35708503
5.
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
6.
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
7.
Glucagon Like Peptide 1 Receptor Agonists for Targeted Delivery of Antisense Oligonucleotides to Pancreatic Beta Cell.
J Am Chem Soc
; 143(9): 3416-3429, 2021 03 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-33626278
8.
Kinetic and subcellular analysis of PS-ASO/protein interactions with P54nrb and RNase H1.
Nucleic Acids Res
; 47(20): 10865-10880, 2019 11 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-31495875
9.
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
10.
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
11.
Enhanced Potency of GalNAc-Conjugated Antisense Oligonucleotides in Hepatocellular Cancer Models.
Mol Ther
; 27(9): 1547-1557, 2019 09 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-31303442
12.
Chemically Modified Cpf1-CRISPR RNAs Mediate Efficient Genome Editing in Mammalian Cells.
Mol Ther
; 26(5): 1228-1240, 2018 05 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-29650467
13.
Asialoglycoprotein receptor 1 mediates productive uptake of N-acetylgalactosamine-conjugated and unconjugated phosphorothioate antisense oligonucleotides into liver hepatocytes.
Nucleic Acids Res
; 45(21): 12388-12400, 2017 Dec 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-29069408
14.
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
15.
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
16.
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
17.
Activating frataxin expression by single-stranded siRNAs targeting the GAA repeat expansion.
Bioorg Med Chem Lett
; 28(17): 2850-2855, 2018 09 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-30076049
18.
Hsp90 protein interacts with phosphorothioate oligonucleotides containing hydrophobic 2'-modifications and enhances antisense activity.
Nucleic Acids Res
; 44(8): 3892-907, 2016 05 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-26945041
19.
Synthetic CRISPR RNA-Cas9-guided genome editing in human cells.
Proc Natl Acad Sci U S A
; 112(51): E7110-7, 2015 Dec 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-26589814
20.
Argonaute 2-dependent Regulation of Gene Expression by Single-stranded miRNA Mimics.
Mol Ther
; 24(5): 946-55, 2016 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-26903376