Detalhe da pesquisa
1.
Chirality matters: stereo-defined phosphorothioate linkages at the termini of small interfering RNAs improve pharmacology in vivo.
Nucleic Acids Res
; 50(3): 1221-1240, 2022 02 22.
Artigo
em Inglês
| MEDLINE | ID: mdl-34268578
2.
From bench to bedside: Improving the clinical safety of GalNAc-siRNA conjugates using seed-pairing destabilization.
Nucleic Acids Res
; 50(12): 6656-6670, 2022 07 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-35736224
3.
Shorter Is Better: The α-(l)-Threofuranosyl Nucleic Acid Modification Improves Stability, Potency, Safety, and Ago2 Binding and Mitigates Off-Target Effects of Small Interfering RNAs.
J Am Chem Soc
; 145(36): 19691-19706, 2023 09 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-37638886
4.
Overcoming GNA/RNA base-pairing limitations using isonucleotides improves the pharmacodynamic activity of ESC+ GalNAc-siRNAs.
Nucleic Acids Res
; 49(19): 10851-10867, 2021 11 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-34648028
5.
Role of a "Magic" Methyl: 2'-Deoxy-2'-α-F-2'-ß-C-methyl Pyrimidine Nucleotides Modulate RNA Interference Activity through Synergy with 5'-Phosphate Mimics and Mitigation of Off-Target Effects.
J Am Chem Soc
; 144(32): 14517-14534, 2022 08 17.
Artigo
em Inglês
| MEDLINE | ID: mdl-35921401
6.
The Nonclinical Disposition and Pharmacokinetic/Pharmacodynamic Properties of N-Acetylgalactosamine-Conjugated Small Interfering RNA Are Highly Predictable and Build Confidence in Translation to Human.
Drug Metab Dispos
; 50(6): 781-797, 2022 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-34154993
7.
Safety evaluation of 2'-deoxy-2'-fluoro nucleotides in GalNAc-siRNA conjugates.
Nucleic Acids Res
; 47(7): 3306-3320, 2019 04 23.
Artigo
em Inglês
| MEDLINE | ID: mdl-30820542
8.
Structural basis for the synergy of 4'- and 2'-modifications on siRNA nuclease resistance, thermal stability and RNAi activity.
Nucleic Acids Res
; 46(16): 8090-8104, 2018 09 19.
Artigo
em Inglês
| MEDLINE | ID: mdl-30107495
9.
Evaluation of GalNAc-siRNA Conjugate Activity in Pre-clinical Animal Models with Reduced Asialoglycoprotein Receptor Expression.
Mol Ther
; 26(1): 105-114, 2018 01 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-28988716
10.
Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc-siRNA conjugates.
Nucleic Acids Res
; 45(19): 10969-10977, 2017 Nov 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-28981809
11.
Chirality Dependent Potency Enhancement and Structural Impact of Glycol Nucleic Acid Modification on siRNA.
J Am Chem Soc
; 139(25): 8537-8546, 2017 06 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-28570818
12.
4'-C-Methoxy-2'-deoxy-2'-fluoro Modified Ribonucleotides Improve Metabolic Stability and Elicit Efficient RNAi-Mediated Gene Silencing.
J Am Chem Soc
; 139(41): 14542-14555, 2017 10 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-28937776
13.
5'-(E)-Vinylphosphonate: A Stable Phosphate Mimic Can Improve the RNAi Activity of siRNA-GalNAc Conjugates.
Chembiochem
; 17(11): 985-9, 2016 Jun 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-27121751
14.
Structural Basis of Duplex Thermodynamic Stability and Enhanced Nuclease Resistance of 5'-C-Methyl Pyrimidine-Modified Oligonucleotides.
J Org Chem
; 81(6): 2261-79, 2016 Mar 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-26940174
15.
Sequence-Defined Oligomers from Hydroxyproline Building Blocks for Parallel Synthesis Applications.
Angew Chem Int Ed Engl
; 55(33): 9529-33, 2016 08 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-27365192
16.
Hepatocyte-specific delivery of siRNAs conjugated to novel non-nucleosidic trivalent N-acetylgalactosamine elicits robust gene silencing in vivo.
Chembiochem
; 16(6): 903-8, 2015 Apr 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-25786782
17.
Multivalent N-acetylgalactosamine-conjugated siRNA localizes in hepatocytes and elicits robust RNAi-mediated gene silencing.
J Am Chem Soc
; 136(49): 16958-61, 2014 Dec 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-25434769
18.
Comprehensive evaluation of canonical versus Dicer-substrate siRNA in vitro and in vivo.
RNA
; 18(3): 557-68, 2012 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-22294662
19.
Biodegradable lipids enabling rapidly eliminated lipid nanoparticles for systemic delivery of RNAi therapeutics.
Mol Ther
; 21(8): 1570-8, 2013 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-23799535
20.
Efficient Downregulation of Alk4 in Skeletal Muscle After Systemic Treatment with Conjugated siRNAs in a Mouse Model for Duchenne Muscular Dystrophy.
Nucleic Acid Ther
; 33(1): 26-34, 2023 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-36269327