Detalhe da pesquisa
1.
CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema.
N Engl J Med
; 390(5): 432-441, 2024 Feb 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-38294975
2.
CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.
N Engl J Med
; 385(6): 493-502, 2021 08 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-34215024
3.
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
4.
Evaluation of RNAi therapeutics VIR-2218 and ALN-HBV for chronic hepatitis B: Results from randomized clinical trials.
J Hepatol
; 79(4): 924-932, 2023 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-37290591
5.
Centyrin ligands for extrahepatic delivery of siRNA.
Mol Ther
; 29(6): 2053-2066, 2021 06 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-33601052
6.
Knockdown of Virus Antigen Expression Increases Therapeutic Vaccine Efficacy in High-Titer Hepatitis B Virus Carrier Mice.
Gastroenterology
; 158(6): 1762-1775.e9, 2020 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-32001321
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.
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
9.
Systematic chemical modifications of single stranded siRNAs significantly improved CTNNB1 mRNA silencing.
Bioorg Med Chem Lett
; 26(18): 4513-4517, 2016 09 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-27503684
10.
Novel endosomolytic poly(amido amine) polymer conjugates for systemic delivery of siRNA to hepatocytes in rodents and nonhuman primates.
Bioconjug Chem
; 25(5): 896-906, 2014 May 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-24742200
11.
Improving the in vivo therapeutic index of siRNA polymer conjugates through increasing pH responsiveness.
Bioconjug Chem
; 25(2): 296-307, 2014 Feb 19.
Artigo
em Inglês
| MEDLINE | ID: mdl-24409989
12.
Endosomolytic bioreducible poly(amido amine disulfide) polymer conjugates for the in vivo systemic delivery of siRNA therapeutics.
Bioconjug Chem
; 24(4): 640-7, 2013 Apr 17.
Artigo
em Inglês
| MEDLINE | ID: mdl-23496378
13.
Induction of apoptosis by an inhibitor of the mitotic kinesin KSP requires both activation of the spindle assembly checkpoint and mitotic slippage.
Cancer Cell
; 8(1): 49-59, 2005 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-16023598
14.
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
15.
Quantitative evaluation of siRNA delivery in vivo.
RNA
; 16(12): 2553-63, 2010 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-20940339
16.
Pyridyl aminothiazoles as potent Chk1 inhibitors: optimization of cellular activity.
Bioorg Med Chem Lett
; 22(7): 2613-9, 2012 Apr 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-22365762
17.
Pyridyl aminothiazoles as potent inhibitors of Chk1 with slow dissociation rates.
Bioorg Med Chem Lett
; 22(7): 2609-12, 2012 Apr 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-22374217
18.
Regulation of microRNA-145 by growth arrest and differentiation.
Exp Cell Res
; 317(4): 488-95, 2011 Feb 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-21111732
19.
Mechanistically probing lipid-siRNA nanoparticle-associated toxicities identifies Jak inhibitors effective in mitigating multifaceted toxic responses.
Mol Ther
; 19(3): 567-75, 2011 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-21179008
20.
Effective siRNA delivery and target mRNA degradation using an amphipathic peptide to facilitate pH-dependent endosomal escape.
Biochem J
; 435(2): 475-87, 2011 Apr 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-21265735