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。Sixteen Different Types of Lipid-Conjugated siRNAs Containing Saturated and Unsaturated Fatty Acids and Exhibiting Enhanced RNAi Potency
Takanori Kubo et al.
ACS CHEMICAL BIOLOGY (2021)
Docosanoic acid conjugation to siRNA enables functional and safe delivery to skeletal and cardiac muscles
Annabelle Biscans et al.
MOLECULAR THERAPY (2021)
A high-throughput Galectin-9 imaging assay for quantifying nanoparticle uptake, endosomal escape and functional RNA delivery
Michael J. Munson et al.
COMMUNICATIONS BIOLOGY (2021)
Imaging small molecule-induced endosomal escape of siRNA
Hampus Du Rietz et al.
NATURE COMMUNICATIONS (2020)
Inclisiran for the Treatment of Heterozygous Familial Hypercholesterolemia
Frederick J. Raal et al.
NEW ENGLAND JOURNAL OF MEDICINE (2020)
Single-Stranded Phosphorothioated Regions Enhance Cellular Uptake of Cholesterol-Conjugated siRNA but Not Silencing Efficacy
Socheata Ly et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2020)
LB002ILLUMINATE-A, A PHASE 3 STUDY OF LUMASIRAN, AN INVESTIGATIONAL RNAI THERAPEUTIC, IN CHILDREN AND ADULTS WITH PRIMARY HYPEROXALURIA TYPE 1 (PH1)
Sander Garrelfs et al.
NEPHROLOGY DIALYSIS TRANSPLANTATION (2020)
The chemical structure and phosphorothioate content of hydrophobically modified siRNAs impact extrahepatic distribution and efficacy
Annabelle Biscans et al.
NUCLEIC ACIDS RESEARCH (2020)
Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria
Eliane Sardh et al.
NEW ENGLAND JOURNAL OF MEDICINE (2019)
The valency of fatty acid conjugates impacts siRNA pharmacokinetics, distribution, and efficacy in vivo
Annabelle Biscans et al.
JOURNAL OF CONTROLLED RELEASE (2019)
Hydrophobicity drives the systemic distribution of lipid-conjugated siRNAs via lipid transport pathways
Maire F. Osborn et al.
NUCLEIC ACIDS RESEARCH (2019)
Diverse lipid conjugates for functional extra-hepatic siRNA delivery in vivo
Annabelle Biscans et al.
NUCLEIC ACIDS RESEARCH (2019)
Advanced siRNA Designs Further Improve In Vivo Performance of GaINAc-siRNA Conjugates
Donald J. Foster et al.
MOLECULAR THERAPY (2018)
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
D. Adams et al.
NEW ENGLAND JOURNAL OF MEDICINE (2018)
Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo
Matthew R. Hassler et al.
NUCLEIC ACIDS RESEARCH (2018)
Improving Small Interfering RNA Delivery In Vivo Through Lipid Conjugation
Maire F. Osborn et al.
NUCLEIC ACID THERAPEUTICS (2018)
RNAi modulation of placental sFLT1 for the treatment of preeclampsia
Anton A. Turanov et al.
NATURE BIOTECHNOLOGY (2018)
Lipid Nanoparticle Systems for Enabling Gene Therapies
Pieter R. Cullis et al.
MOLECULAR THERAPY (2017)
The chemical evolution of oligonucleotide therapies of clinical utility
Anastasia Khvorova et al.
NATURE BIOTECHNOLOGY (2017)
5′-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo
Reka A. Haraszti et al.
NUCLEIC ACIDS RESEARCH (2017)
Lipophilic siRNA targets albumin in situ and promotes bioavailability, tumor penetration, and carrier-free gene silencing
Samantha M. Sarett et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2017)
Pharmacokinetic Profiling of Conjugated Therapeutic Oligonucleotides: A High-Throughput Method Based Upon Serial Blood Microsampling Coupled to Peptide Nucleic Acid Hybridization Assay
Bruno M. D. C. Godinho et al.
NUCLEIC ACID THERAPEUTICS (2017)
Preclinical and Clinical Advances of GalNAc-Decorated Nucleic Acid Therapeutics
Yuanyu Huang
MOLECULAR THERAPY-NUCLEIC ACIDS (2017)
9 5′-(E)-Vinylphosphonate: A Stable Phosphate Mimic Can Improve the RNAi Activity of siRNA-GalNAc Conjugates
Rubina Parmar et al.
CHEMBIOCHEM (2016)
A High-Throughput Method for Direct Detection of Therapeutic Oligonucleotide-Induced Gene Silencing In Vivo
Andrew H. Coles et al.
NUCLEIC ACID THERAPEUTICS (2016)
Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides
Richard S. Geary et al.
ADVANCED DRUG DELIVERY REVIEWS (2015)
Hepatocyte-Specific Delivery of siRNAs Conjugated to Novel Non-nucleosidic Trivalent N-Acetylgalactosamine Elicits Robust Gene Silencing in Vivo
Kallanthottathil G. Rajeev et al.
CHEMBIOCHEM (2015)
Visualizing lipid-formulated siRNA release from endosomes and target gene knockdown
Anders Wittrup et al.
NATURE BIOTECHNOLOGY (2015)
Plasmodium berghei ANKA causes intestinal malaria associated with dysbiosis
Tomoyo Taniguchi et al.
SCIENTIFIC REPORTS (2015)
Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing
Jayaprakash K. Nair et al.
JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2014)
The rough endoplasmatic reticulum is a central nucleation site of siRNA-mediated RNA silencing
Lukas Stalder et al.
EMBO JOURNAL (2013)
Image-based analysis of lipid nanoparticle-mediated siRNA delivery, intracellular trafficking and endosomal escape
Jerome Gilleron et al.
NATURE BIOTECHNOLOGY (2013)
Advances in Lipid Nanoparticles for siRNA Delivery
Yuen Tam et al.
Pharmaceutics (2013)
Maximizing the Potency of siRNA Lipid Nanoparticles for Hepatic Gene Silencing In Vivo
Muthusamy Jayaraman et al.
ANGEWANDTE CHEMIE-INTERNATIONAL EDITION (2012)
Galectin 8 targets damaged vesicles for autophagy to defend cells against bacterial invasion
Teresa L. M. Thurston et al.
NATURE (2012)
Fiji: an open-source platform for biological-image analysis
Johannes Schindelin et al.
NATURE METHODS (2012)
NIH Image to ImageJ: 25 years of image analysis
Caroline A. Schneider et al.
NATURE METHODS (2012)
Rational design of cationic lipids for siRNA delivery
Sean C. Semple et al.
NATURE BIOTECHNOLOGY (2010)
Mechanisms of Endocytosis
Gary J. Doherty et al.
ANNUAL REVIEW OF BIOCHEMISTRY (2009)
Efficient in vivo delivery of siRNA to the liver by conjugation of α-tocopherol
Kazutaka Nishina et al.
MOLECULAR THERAPY (2008)
Nucleoside modifications modulate activation of the protein kinase PKR in an RNA structure-specific manner
Subba Rao Nallagatla et al.
RNA (2008)
Effects of cholesterol-tagged small interfering RNAs targeting 12/15-lipoxygenase on parameters of diabetic nephropathy in a mouse model of type 1 diabetes
Hang Yuan et al.
AMERICAN JOURNAL OF PHYSIOLOGY-RENAL PHYSIOLOGY (2008)
Mechanisms and optimization of in vivo delivery of lipophilic siRNAs
Christian Wolfrum et al.
NATURE BIOTECHNOLOGY (2007)
Forty mouse strain survey of body composition
Danielle R. Reed et al.
PHYSIOLOGY & BEHAVIOR (2007)
Position-specific chemical modification of siRNAs reduces off-target'' transcript silencing
Aimee L. Jackson et al.
RNA (2006)
Fully 2′-modified oligonucleotide duplexes with improved in vitro potency and stability compared to unmodified small interfering RNA
CR Allerson et al.
JOURNAL OF MEDICINAL CHEMISTRY (2005)
Rational siRNA design for RNA interference
A Reynolds et al.
NATURE BIOTECHNOLOGY (2004)
Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs
J Soutschek et al.
NATURE (2004)
分享论文
