标题
Ligand conjugate SAR and enhanced delivery in NHP
作者
关键词
ASGPr, GalNAc, siRNA, endosomal release
出版物
MOLECULAR THERAPY
Volume -, Issue -, Pages -
出版商
Elsevier BV
发表日期
2021-06-04
DOI
10.1016/j.ymthe.2021.06.002
参考文献
相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。- Delivery of Oligonucleotides to the Liver with GalNAc: From Research to Registered Therapeutic Drug
- (2020) Alexandre J. Debacker et al. MOLECULAR THERAPY
- Investigating the pharmacodynamic durability of GalNAc–siRNA conjugates
- (2020) Christopher R Brown et al. NUCLEIC ACIDS RESEARCH
- An Open‐Label, Multicenter, Phase I, Dose Escalation Study with Phase II Expansion Cohort to Determine the Safety, Pharmacokinetics, and Preliminary Antitumor Activity of Intravenous TKM‐080301 in Subjects with Advanced Hepatocellular Carcinoma
- (2019) Imane El Dika et al. ONCOLOGIST
- Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria
- (2019) Eliane Sardh et al. NEW ENGLAND JOURNAL OF MEDICINE
- Clinical advances of siRNA therapeutics
- (2019) Bo Hu et al. JOURNAL OF GENE MEDICINE
- Current Aspects of siRNA Bioconjugate for In Vitro and In Vivo Delivery
- (2019) Wanyi Tai MOLECULES
- Targeted mRNA Therapy for Ornithine Transcarbamylase Deficiency
- (2018) Mary G. Prieve et al. MOLECULAR THERAPY
- Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
- (2018) David Adams et al. NEW ENGLAND JOURNAL OF MEDICINE
- GalNAc-siRNA Conjugates: Leading the Way for Delivery of RNAi Therapeutics
- (2018) Aaron D. Springer et al. Nucleic Acid Therapeutics
- Co-Injection of a Targeted, Reversibly Masked Endosomolytic Polymer Dramatically Improves the Efficacy of Cholesterol-Conjugated Small Interfering RNAs In Vivo
- (2018) So C. Wong et al. Nucleic Acid Therapeutics
- An investigational RNAi therapeutic targeting Factor XII (ALN-F12) for the treatment of hereditary angioedema
- (2018) Jingxuan Liu et al. RNA
- Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc–siRNA conjugates
- (2017) Jayaprakash K. Nair et al. NUCLEIC ACIDS RESEARCH
- Characterizing the effect of GalNAc and phosphorothioate backbone on binding of antisense oligonucleotides to the asialoglycoprotein receptor
- (2017) Karsten Schmidt et al. NUCLEIC ACIDS RESEARCH
- Well-Defined Multivalent Ligands for Hepatocytes Targeting via Asialoglycoprotein Receptor
- (2016) Xiangang Huang et al. BIOCONJUGATE CHEMISTRY
- An Investigational RNAi Therapeutic Targeting Glycolate Oxidase Reduces Oxalate Production in Models of Primary Hyperoxaluria
- (2016) Abigail Liebow et al. JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY
- Disposition and Pharmacology of a GalNAc3-conjugated ASO Targeting Human Lipoprotein (a) in Mice
- (2016) Rosie Z Yu et al. Molecular Therapy-Nucleic Acids
- siRNA Conjugates Carrying Sequentially Assembled Trivalent N-Acetylgalactosamine Linked Through Nucleosides Elicit Robust Gene Silencing In Vivo in Hepatocytes
- (2015) Shigeo Matsuda et al. ACS Chemical Biology
- An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia
- (2015) Alfica Sehgal et al. NATURE MEDICINE
- Preclinical Development of a Subcutaneous ALAS1 RNAi Therapeutic for Treatment of Hepatic Porphyrias Using Circulating RNA Quantification
- (2015) Amy Chan et al. Molecular Therapy-Nucleic Acids
- Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing
- (2014) Jayaprakash K. Nair et al. JOURNAL OF THE AMERICAN CHEMICAL SOCIETY
- Targeted delivery of antisense oligonucleotides to hepatocytes using triantennaryN-acetyl galactosamine improves potency 10-fold in mice
- (2014) Thazha P. Prakash et al. NUCLEIC ACIDS RESEARCH
- Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial
- (2013) Kevin Fitzgerald et al. LANCET
- Glycomimetic Ligands for the Human Asialoglycoprotein Receptor
- (2012) Sreeman K. Mamidyala et al. JOURNAL OF THE AMERICAN CHEMICAL SOCIETY
- Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms
- (2010) Akin Akinc et al. MOLECULAR THERAPY
- Lipid-like materials for low-dose, in vivo gene silencing
- (2010) K. T. Love et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
Find Funding. Review Successful Grants.
Explore over 25,000 new funding opportunities and over 6,000,000 successful grants.
ExploreFind the ideal target journal for your manuscript
Explore over 38,000 international journals covering a vast array of academic fields.
Search