Detalhe da pesquisa
1.
RNAi-mediated reduction of hepatic Tmprss6 diminishes anemia and secondary iron overload in a splenectomized mouse model of ß-thalassemia intermedia.
Am J Hematol
; 93(6): 745-750, 2018 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-29498084
2.
An Investigational RNAi Therapeutic Targeting Glycolate Oxidase Reduces Oxalate Production in Models of Primary Hyperoxaluria.
J Am Soc Nephrol
; 28(2): 494-503, 2017 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-27432743
3.
Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates.
Proc Natl Acad Sci U S A
; 111(11): 3955-60, 2014 Mar 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-24516150
4.
An RNAi therapeutic targeting Tmprss6 decreases iron overload in Hfe(-/-) mice and ameliorates anemia and iron overload in murine ß-thalassemia intermedia.
Blood
; 121(7): 1200-8, 2013 Feb 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-23223430
5.
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
6.
Harnessing a physiologic mechanism for siRNA delivery with mimetic lipoprotein particles.
Mol Ther
; 20(8): 1582-9, 2012 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-22850721
7.
Nonclinical Safety Profile of Revusiran, a 1st-Generation GalNAc-siRNA Conjugate for Treatment of Hereditary Transthyretin-Mediated Amyloidosis.
Nucleic Acid Ther
; 30(1): 33-49, 2020 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-31821125
8.
An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia.
Nat Med
; 21(5): 492-7, 2015 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-25849132