An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia.
about
New developments in the management of moderate-to-severe hemophilia BsiRNA Versus miRNA as Therapeutics for Gene SilencingTherapeutic Strategies and New Intervention Points in Chronic Hepatitis Delta Virus InfectionThe current state of adverse event reporting in hemophilia.Visualization of self-delivering hydrophobically modified siRNA cellular internalization.siRNA carrying an (E)-vinylphosphonate moiety at the 5΄ end of the guide strand augments gene silencing by enhanced binding to human Argonaute-2.Hemophilia Care in the Pediatric Age.The notorious R.N.A. in the spotlight - drug or target for the treatment of diseaseCRISPR/Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice.Design and characterization of an APC-specific serpin for the treatment of hemophilia.Global measurement of coagulation in plasma from normal and haemophilia dogs using a novel modified thrombin generation test - Demonstrated in vitro and ex vivo.Stabilin-1 and Stabilin-2 are specific receptors for the cellular internalization of phosphorothioate-modified antisense oligonucleotides (ASOs) in the liver.Preclinical evaluation of RNAi as a treatment for transthyretin-mediated amyloidosis.Preclinical and Clinical Advances of GalNAc-Decorated Nucleic Acid Therapeutics.New and Emerging Agents for the Treatment of Hemophilia: Focus on Extended Half-Life Recombinant Clotting Proteins.New products for the treatment of haemophilia.Identification and function probing of an antithrombin IIIβ conformation-specific antibody.Tailoring hemostatic therapies to lower inhibitor development in previously untreated patients with severe hemophilia A.Haemophilia treatment in 2030.The utility of thromboelastography in inherited and acquired bleeding disorders.Rethinking events in the haemostatic process: role of factor V and TFPI.Emerging drugs for the treatment of hemophilia A and B.2017 Clinical trials update: Innovations in hemophilia therapy.The chemical evolution of oligonucleotide therapies of clinical utility.Oligonucleotides targeting coagulation factor mRNAs: use in thrombosis and hemophilia research and therapy.Oral nucleic acid therapy using multicompartmental delivery systems.An Investigational RNAi Therapeutic Targeting Glycolate Oxidase Reduces Oxalate Production in Models of Primary Hyperoxaluria.Extended half-life clotting factor concentrates: results from published clinical trials.Structural Analysis of Human Argonaute-2 Bound to a Modified siRNA Guide.5΄-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo.Harnessing human ADAR2 for RNA repair - Recoding a PINK1 mutation rescues mitophagy.The second Team Haemophilia Education Meeting, 2016, Frankfurt, Germany.Partnering to change the world for people with haemophilia: 6(th) Haemophilia Global Summit, Prague, Czech Republic, 24-26(th) September 2015.Alternative therapies for the management of inhibitors.Novel approaches to hemophilia therapy: successes and challenges.Future of Haemophilia Research in India.Thrombin generation and other coagulation parameters in a patient with homozygous congenital protein S deficiency on treatment with rivaroxaban.Induction of RNAi Responses by Short Left-Handed Hairpin RNAi Triggers.Evaluation of GalNAc-siRNA Conjugate Activity in Pre-clinical Animal Models with Reduced Asialoglycoprotein Receptor Expression.Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc-siRNA conjugates.
P2860
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P2860
An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia.
description
2015 nî lūn-bûn
@nan
2015年の論文
@ja
2015年学术文章
@wuu
2015年学术文章
@zh
2015年学术文章
@zh-cn
2015年学术文章
@zh-hans
2015年学术文章
@zh-my
2015年学术文章
@zh-sg
2015年學術文章
@yue
2015年學術文章
@zh-hant
name
An RNAi therapeutic targeting ...... mote hemostasis in hemophilia.
@en
An RNAi therapeutic targeting ...... mote hemostasis in hemophilia.
@nl
type
label
An RNAi therapeutic targeting ...... mote hemostasis in hemophilia.
@en
An RNAi therapeutic targeting ...... mote hemostasis in hemophilia.
@nl
prefLabel
An RNAi therapeutic targeting ...... mote hemostasis in hemophilia.
@en
An RNAi therapeutic targeting ...... mote hemostasis in hemophilia.
@nl
P2093
P2860
P356
P1433
P1476
An RNAi therapeutic targeting ...... omote hemostasis in hemophilia
@en
P2093
Akin Akinc
Alexander V Kel'in
Alfica Sehgal
Amy R Simon
Benny Sorensen
Brian Cooley
Don Foster
Harsha Prabhala
Husain Attarwala
Jayaprakash K Nair
P2860
P2888
P304
P356
10.1038/NM.3847
P407
P577
2015-04-13T00:00:00Z
P6179
1014623450