4.5 Review

Therapeutic siRNA: State-of-the-Art and Future Perspectives

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。
Article Biochemistry & Molecular Biology

Specific delivering of RNAi using Spike's aptamer-functionalized lipid nanoparticles for targeting SARS-CoV-2: A strong anti-Covid drug in a clinical case study

Haidar Saify Nabiabad et al.

Summary: The study utilized aptamer-functionalized lipid nanoparticles (LNPs) to deliver RNAi molecules for treating SARS-CoV-2 infection, showing promising results. The aptamer in the nanocarrier serves two crucial functions - delivering the drug to the cell surface and inhibiting virus entry by binding to the virus.

CHEMICAL BIOLOGY & DRUG DESIGN (2022)

Article Medicine, Research & Experimental

A siRNA targets and inhibits a broad range of SARS-CoV-2 infections including Delta variant

Yi-Chung Chang et al.

Summary: The inhalable siRNA drug C6G25S, covering 99.8% of SARS-CoV-2 variants, shows effectiveness in inhibiting dominant strains and preventing lung damage in animal models.

EMBO MOLECULAR MEDICINE (2022)

Article Gastroenterology & Hepatology

Efficacy and safety of givosiran for acute hepatic porphyria: 24-month interim analysis of the randomized phase 3 ENVISION study

Paolo Ventura et al.

Summary: The study showed that long-term use of givosiran can significantly reduce attack frequency, hemin use, and severity of daily worst pain in patients with acute hepatic porphyria, while also improving quality of life.

LIVER INTERNATIONAL (2022)

Article Biochemistry & Molecular Biology

The UCSC Genome Browser database: 2022 update

Brian T. Lee et al.

Summary: The UCSC Genome Browser is a graphical viewer for exploring genome annotations, providing integrated tools for visualizing, comparing, analyzing, and sharing genomic datasets. Updates this year include new public hub assemblies for new organisms, updated clinical tracks, a new Track Sets feature, enhanced variant displays, and a tool for placing new SARS-CoV-2 genomes in a global phylogenetic tree. Other improvements focus on usability features such as informative mouseover displays and new fonts.

NUCLEIC ACIDS RESEARCH (2022)

Review Pharmacology & Pharmacy

Regulatory guidelines and preclinical tools to study the biodistribution of RNA therapeutics

P. Vervaeke et al.

Summary: The success of mRNA-based COVID-19 vaccines signals a new chapter in modern medicine, but the regulatory framework for mRNA therapeutics lags behind. This review discusses guidelines for preclinical biodistribution studies of mRNA/siRNA therapeutics and highlights the differences for mRNA vaccines. The study also explores the role of in vivo RNA imaging techniques and other assays in meeting regulatory requirements.

ADVANCED DRUG DELIVERY REVIEWS (2022)

Article Surgery

Patisiran treatment in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy after liver transplantation

Hartmut H. Schmidt et al.

Summary: This study evaluated the efficacy and safety of patisiran in patients with ATTRv amyloidosis with polyneuropathy progression following liver transplantation. The results showed that patisiran reduced serum TTR levels, improved neuropathy and quality of life, and stabilized disability and nutritional status. Most patients had normal liver function. Patisiran treatment was well tolerated.

AMERICAN JOURNAL OF TRANSPLANTATION (2022)

Article Urology & Nephrology

Safety, pharmacodynamics, and exposure-response modeling results from a first-in-human phase 1 study of nedosiran (PHYOX1) in primary hyperoxaluria

Bernd Hoppe et al.

Summary: This study evaluated the safety, pharmacokinetics, pharmacodynamics, and dose-response relationship of Nedosiran in healthy individuals and patients with PH. The results showed that single-dose Nedosiran demonstrated acceptable safety and evidence of pharmacodynamic effect in both PH1 and PH2 subpopulations.

KIDNEY INTERNATIONAL (2022)

Article Multidisciplinary Sciences

Identification of the toxic 6mer seed consensus for human cancer cells

Monal Patel et al.

Summary: 6mer seed toxicity is a novel approach to kill cancer cells by triggering cell death mechanism. This approach utilizes siRNAs or shRNAs with a specific G-rich nucleotide composition to recognize and eliminate cancer cells in cell lines. The study reveals that GGGGGC consensus is the most toxic average 6mer seed for human cancer cells.

SCIENTIFIC REPORTS (2022)

Article Biotechnology & Applied Microbiology

Selection and Validation of siRNAs Preventing Uptake and Replication of SARS-CoV-2

Maik Friedrich et al.

Summary: In 2019, the SARS-CoV-2 outbreak caused a global pandemic. Besides vaccines, there is a need for novel treatment options. The use of siRNA targeting ACE2 and the SARS-CoV-2 RNA genome is an effective strategy to prevent viral entry into host cells or interfere with viral replication.

FRONTIERS IN BIOENGINEERING AND BIOTECHNOLOGY (2022)

Article Pharmacology & Pharmacy

Single-Dose Pharmacokinetics and Pharmacodynamics of Transthyretin Targeting N-acetylgalactosamine-Small Interfering Ribonucleic Acid Conjugate, Vutrisiran, in Healthy Subjects

Bahru A. Habtemariam et al.

Summary: Vutrisiran demonstrated potent and sustained reduction of TTR in healthy subjects, with favorable pharmacokinetics and safety profile. The results were similar in Japanese and non-Japanese subjects.

CLINICAL PHARMACOLOGY & THERAPEUTICS (2021)

Article Pharmacology & Pharmacy

Pharmacokinetic and Pharmacodynamic Properties of Cemdisiran, an RNAi Therapeutic Targeting Complement Component 5, in Healthy Subjects and Patients with Paroxysmal Nocturnal Hemoglobinuria

Prajakta Badri et al.

Summary: The study evaluated the safety and pharmacodynamics of cemdisiran in healthy subjects and PNH patients, showing that cemdisiran effectively suppressed C5 protein and maintained stability even after multiple doses.

CLINICAL PHARMACOKINETICS (2021)

Article Biochemistry & Molecular Biology

Database resources of the National Center for Biotechnology Information

Eric W. Sayers et al.

Summary: The National Center for Biotechnology Information (NCBI) offers a wide range of online resources for biological information and data, including GenBank (R) nucleic acid sequence database and PubMed (R) database of citations and abstracts. Entrez system allows search and retrieval operations for most of these data, with E-utilities serving as the programming interface. Additional resources such as PMC, Bookshelf, Genome Data Viewer, SRA, ClinVar, dbSNP, dbVar, Pathogen Detection, BLAST, Primer-BLAST, IgBLAST, iCn3D, and PubChem are also accessible through the NCBI home page.

NUCLEIC ACIDS RESEARCH (2021)

Article Pharmacology & Pharmacy

Lumasiran: First Approval

Lesley J. Scott et al.

Summary: Lumasiran is a drug developed for the treatment of primary hyperoxaluria type 1 by targeting a specific gene to inhibit the synthesis of toxic metabolites. It received approvals in the EU and USA, marking its global recognition.
Review Cell Biology

Gene regulation by long non-coding RNAs and its biological functions

Luisa Statello et al.

Summary: Recent studies have shed new light on the biogenesis and functions of long non-coding RNAs (lncRNAs), showcasing their diverse roles in gene regulation and signaling pathways, particularly in the contexts of neuronal disorders, immune responses, and cancer. The discovery of unique biogenesis pathways and subcellular localizations of lncRNAs has opened up potential therapeutic avenues for targeting lncRNAs in various biological and pathophysiological conditions.

NATURE REVIEWS MOLECULAR CELL BIOLOGY (2021)

Article Hematology

Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran-Results of the phase 1 inhibitor cohort

K. John Pasi et al.

Summary: Fitusiran, an investigational small interfering RNA therapy, was well tolerated and effectively reduced antithrombin levels in patients with hemophilia A or B with inhibitors. This monthly treatment regimen may reduce bleeding episodes and improve quality of life.

JOURNAL OF THROMBOSIS AND HAEMOSTASIS (2021)

Article Cell Biology

A first-in-human phase 0 clinical study of RNA interference-based spherical nucleic acids in patients with recurrent glioblastoma

Priya Kumthekar et al.

Summary: The research team has developed a novel treatment approach for GBM using SNAs for precision medicine treatment, which has shown potential in clinical trials.

SCIENCE TRANSLATIONAL MEDICINE (2021)

Article Allergy

Silencing of SARS-CoV-2 with modified siRNA-peptide dendrimer formulation

Musa Khaitov et al.

Summary: The study identified siR-7 as the most efficient siRNA targeting SARS-CoV-2 RNA-dependent RNA polymerase in inhibiting viral replication in vitro. Modification with LNAs and complexation with the designed peptide dendrimer enhanced the antiviral capacity of siR-7 in vitro. Inhalation of siR-7-EM/KK-46 led to significant reduction of virus titer and lung inflammation in vivo, demonstrating its potential as a therapeutic strategy for COVID-19 patients.

ALLERGY (2021)

Article Urology & Nephrology

Phase 1/2 Study of Lumasiran for Treatment of Primary Hyperoxaluria Type 1 A Placebo-Controlled Randomized Clinical Trial

Yaacov Frishberg et al.

Summary: This study evaluated the safety and pharmacodynamic characteristics of Lumasiran in patients with primary hyperoxaluria type 1, showing that Lumasiran has an acceptable safety profile and can reduce urinary oxalate excretion in patients to near-normal levels.

CLINICAL JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY (2021)

Review Pharmacology & Pharmacy

Givosiran: A Review in Acute Hepatic Porphyria

Yahiya Y. Syed

Summary: Givosiran is an approved treatment for AHP, significantly reducing attack rates and improving symptoms in patients. Despite a risk of adverse events, it offers the convenience of subcutaneous administration and is an important new therapeutic option for AHP patients with severe recurrent attacks.
Article Biotechnology & Applied Microbiology

A SARS-CoV-2 targeted siRNA-nanoparticle therapy for COVID-19

Adi Idris et al.

Summary: This study presents a highly effective siRNA therapeutic against SARS-CoV-2 infection using a novel LNP delivery system, with the potential to significantly inhibit the virus in the lungs and provide a survival advantage to treated mice.

MOLECULAR THERAPY (2021)

Article Medicine, General & Internal

Lumasiran, an RNAi Therapeutic for Primary Hyperoxaluria Type 1

Sander F. Garrelfs et al.

Summary: Primary hyperoxaluria type 1 is a rare genetic disease caused by hepatic overproduction of oxalate, leading to kidney stones, nephrocalcinosis, kidney failure, and systemic oxalosis. This trial tested the efficacy of lumasiran, an RNAi therapeutic agent, in reducing hepatic oxalate production. The results showed that lumasiran significantly reduced urinary oxalate excretion, alleviating the cause of kidney failure in PH1 patients.

NEW ENGLAND JOURNAL OF MEDICINE (2021)

Article Cardiac & Cardiovascular Systems

Teprasiran, a Small Interfering RNA, for the Prevention of Acute Kidney Injury in High-Risk Patients Undergoing Cardiac Surgery A Randomized Clinical Study

Matthias Thielmann et al.

Summary: In high-risk patients undergoing cardiac surgery, a single 10 mg/kg dose of teprasiran significantly reduced the incidence, severity, and duration of early AKI compared to placebo.

CIRCULATION (2021)

Article Biochemistry & Molecular Biology

Ensembl 2021

Kevin L. Howe et al.

Summary: The Ensembl project provides genome annotation and data dissemination services for vertebrate species, including detailed annotation of gene structures, regulatory elements, and variants, as well as inferring the evolutionary history of genes and genomes. They offer integrated genomic data through various means such as genome browsers, search interfaces, specialist tools, and download files. Recent developments include the Ensembl Rapid Release and the SARS-CoV-2 genome browser, aiding in the international scientific response to the COVID-19 pandemic.

NUCLEIC ACIDS RESEARCH (2021)

Review Pharmacology & Pharmacy

Inclisiran-New hope in the management of lipid disorders?

Krzysztof Dyrbus et al.

JOURNAL OF CLINICAL LIPIDOLOGY (2020)

Article Pharmacology & Pharmacy

Givosiran: First Approval

Lesley J. Scott

Article Chemistry, Multidisciplinary

Extracellular vesicle (ECV)-modified polyethylenimine (PEI) complexes for enhanced siRNA delivery in vitro and in vivo

Petro Zhupanyn et al.

JOURNAL OF CONTROLLED RELEASE (2020)

Article Multidisciplinary Sciences

Imaging small molecule-induced endosomal escape of siRNA

Hampus Du Rietz et al.

NATURE COMMUNICATIONS (2020)

Article Medicine, General & Internal

Two Phase 3 Trials of Inclisiran in Patients with Elevated LDL Cholesterol

Kausik K. Ray et al.

NEW ENGLAND JOURNAL OF MEDICINE (2020)

Article Medicine, General & Internal

Inclisiran for the Treatment of Heterozygous Familial Hypercholesterolemia

Frederick J. Raal et al.

NEW ENGLAND JOURNAL OF MEDICINE (2020)

Article Biochemistry & Molecular Biology

Phosphorothioate modified oligonucleotide-protein interactions

Stanley T. Crooke et al.

NUCLEIC ACIDS RESEARCH (2020)

Article Medicine, Research & Experimental

Development of an RNAi therapeutic for alpha-1-antitrypsin liver disease

Christine I. Wooddell et al.

JCI INSIGHT (2020)

Review Biochemistry & Molecular Biology

Therapeutic siRNA: state of the art

Bo Hu et al.

SIGNAL TRANSDUCTION AND TARGETED THERAPY (2020)

Review Chemistry, Medicinal

2019 FDA TIDES (Peptides and Oligonucleotides) Harvest

Danah Al Shaer et al.

PHARMACEUTICALS (2020)

Review Immunology

RIG-I-like receptors: their regulation and roles in RNA sensing

Jan Rehwinkel et al.

NATURE REVIEWS IMMUNOLOGY (2020)

Review Medicine, Research & Experimental

Therapeutic Antisense Oligonucleotides Are Coming of Age

C. Frank Bennett

ANNUAL REVIEW OF MEDICINE, VOL 70 (2019)

Article Biochemistry & Molecular Biology

Fructose-induced hypertriglyceridemia in rhesus macaques is attenuated with fish oil or ApoC3 RNA interference

Andrew A. Butler et al.

JOURNAL OF LIPID RESEARCH (2019)

Review Biotechnology & Applied Microbiology

The current state and future directions of RNAi-based therapeutics

Ryan L. Setten et al.

NATURE REVIEWS DRUG DISCOVERY (2019)

Article Medicine, General & Internal

Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria

Eliane Sardh et al.

NEW ENGLAND JOURNAL OF MEDICINE (2019)

Article Biochemistry & Molecular Biology

Safety evaluation of 2-deoxy-2-fluoro nucleotides in GalNAc-siRNA conjugates

Maja M. Janas et al.

NUCLEIC ACIDS RESEARCH (2019)

Review Pharmacology & Pharmacy

Strategies, design, and chemistry in siRNA delivery systems

Yizhou Dong et al.

ADVANCED DRUG DELIVERY REVIEWS (2019)

Article Biotechnology & Applied Microbiology

Chemical modification of PS-ASO therapeutics reduces cellular protein-binding and improves the therapeutic index

Wen Shen et al.

NATURE BIOTECHNOLOGY (2019)

Article Multidisciplinary Sciences

Regulating intracellular fate of siRNA by endoplasmic reticulum membrane-decorated hybrid nanoplexes

Chong Qiu et al.

NATURE COMMUNICATIONS (2019)

Review Chemistry, Multidisciplinary

Rekindling RNAi Therapy: Materials Design Requirements for In Vivo siRNA Delivery

Byungji Kim et al.

ADVANCED MATERIALS (2019)

Editorial Material Biotechnology & Applied Microbiology

Perspectives, issues and solutions in RNAi therapy: the expected and the less expected

Achim Aigner

NANOMEDICINE (2019)

News Item Biotechnology & Applied Microbiology

PCSK9-gene-silencing, cholesterol-lowering drug impresses

Cormac Sheridan

NATURE BIOTECHNOLOGY (2019)

Review Gastroenterology & Hepatology

Acute Hepatic Porphyrias: Review and Recent Progress

Bruce Wang et al.

HEPATOLOGY COMMUNICATIONS (2019)

Review Chemistry, Medicinal

2018 FDA Tides Harvest

Danah Al Shaer et al.

PHARMACEUTICALS (2019)

Article Biochemistry & Molecular Biology

Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs

Xiulong Shen et al.

NUCLEIC ACIDS RESEARCH (2018)

Editorial Material Pharmacology & Pharmacy

Tivanisiran, a novel siRNA for the treatment of dry eye disease

Javier Moreno-Montanes et al.

EXPERT OPINION ON INVESTIGATIONAL DRUGS (2018)

Review Biochemistry & Molecular Biology

Noncoding RNAs in disease

Evangelia Lekka et al.

FEBS LETTERS (2018)

Article Biotechnology & Applied Microbiology

Advanced siRNA Designs Further Improve In Vivo Performance of GaINAc-siRNA Conjugates

Donald J. Foster et al.

MOLECULAR THERAPY (2018)

Article Medicine, General & Internal

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

D. Adams et al.

NEW ENGLAND JOURNAL OF MEDICINE (2018)

Review Biochemistry & Molecular Biology

GalNAc-siRNA Conjugates: Leading the Way for Delivery of RNAi Therapeutics

Aaron D. Springer et al.

NUCLEIC ACID THERAPEUTICS (2018)

Article Medicine, Research & Experimental

Liver-Targeted Anti-HBV Single-Stranded Oligonucleotides with Locked Nucleic Acid Potently Reduce HBV Gene Expression In Vivo

Hassan Javanbakht et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2018)

Review Biotechnology & Applied Microbiology

Long noncoding RNAs: regulation, function and cancer

Aras Rafiee et al.

BIOTECHNOLOGY AND GENETIC ENGINEERING REVIEWS, VOL 34, ISSUE 2 (2018)

Article Pharmacology & Pharmacy

Patisiran: First Global Approval

Sheridan M. Hoy

Article Multidisciplinary Sciences

Exosomes facilitate therapeutic targeting of oncogenic KRAS in pancreatic cancer

Sushrut Kamerkar et al.

NATURE (2017)

Review Biotechnology & Applied Microbiology

The chemical evolution of oligonucleotide therapies of clinical utility

Anastasia Khvorova et al.

NATURE BIOTECHNOLOGY (2017)

Article Biotechnology & Applied Microbiology

Overcoming cellular barriers for RNA therapeutics

Steven F. Dowdy

NATURE BIOTECHNOLOGY (2017)

Article Medicine, General & Internal

Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy

K. J. Pasi et al.

NEW ENGLAND JOURNAL OF MEDICINE (2017)

Article Medicine, General & Internal

A Highly Durable RNAi Therapeutic Inhibitor of PCSK9

Kevin Fitzgerald et al.

NEW ENGLAND JOURNAL OF MEDICINE (2017)

Article Biochemistry & Molecular Biology

5′-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo

Reka A. Haraszti et al.

NUCLEIC ACIDS RESEARCH (2017)

Article Pharmacology & Pharmacy

Strategies for Improving siRNA-Induced Gene Silencing Efficiency

Fatemeh Safari et al.

ADVANCED PHARMACEUTICAL BULLETIN (2017)

Review Biotechnology & Applied Microbiology

Precise and efficient siRNA design: a key point in competent gene silencing

E. Fakhr et al.

CANCER GENE THERAPY (2016)

Review Multidisciplinary Sciences

From the RNA world to the clinic

Bruce A. Sullenger et al.

SCIENCE (2016)

Article Ophthalmology

Safety and Efficacy Clinical Trials for SYL1001, a Novel Short Interfering RNA for the Treatment of Dry Eye Disease

Jose Manuel Benitez-Del-Castillo et al.

INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE (2016)

Review Chemistry, Multidisciplinary

Nanomedicine in cancer therapy: Challenges, opportunities, and clinical applications

Andreas Wicki et al.

JOURNAL OF CONTROLLED RELEASE (2015)

Article Biotechnology & Applied Microbiology

Visualizing lipid-formulated siRNA release from endosomes and target gene knockdown

Anders Wittrup et al.

NATURE BIOTECHNOLOGY (2015)

Review Medicine, Research & Experimental

siRNA Versus miRNA as Therapeutics for Gene Silencing

Jenny K. W. Lam et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2015)

Article Biochemistry & Molecular Biology

Base Modification Strategies to Modulate Immune Stimulation by an siRNA

Rachel Anne P. Valenzuela et al.

CHEMBIOCHEM (2015)

Review Biochemistry & Molecular Biology

Toll-Like Receptors in Antiviral Innate Immunity

Sandra N. Lester et al.

JOURNAL OF MOLECULAR BIOLOGY (2014)

Article Multidisciplinary Sciences

RNAi-mediated silencing of hepatic Alas1 effectively prevents and treats the induced acute attacks in acute intermittent porphyria mice

Makiko Yasuda et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2014)

Article Biotechnology & Applied Microbiology

Efficiency of siRNA delivery by lipid nanoparticles is limited by endocytic recycling

Gaurav Sahay et al.

NATURE BIOTECHNOLOGY (2013)

Review Chemistry, Physical

Delivery materials for siRNA therapeutics

Rosemary Kanasty et al.

NATURE MATERIALS (2013)

Article Medicine, General & Internal

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

Teresa Coelho et al.

NEW ENGLAND JOURNAL OF MEDICINE (2013)

Article Biochemistry & Molecular Biology

Development of modified siRNA molecules incorporating 5-fluoro-2'-deoxyuridine residues to enhance cytotoxicity

Shao-yu Wu et al.

NUCLEIC ACIDS RESEARCH (2013)

Review Urology & Nephrology

Overview of Complement Activation and Regulation

Marina Noris et al.

SEMINARS IN NEPHROLOGY (2013)

Article Medicine, Research & Experimental

Influence of Polyethylene Glycol Lipid Desorption Rates on Pharmacokinetics and Pharmacodynamics of siRNA Lipid Nanoparticles

Barbara L. Mui et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2013)

Article Chemistry, Multidisciplinary

In Vitro-In Vivo Translation of Lipid Nanoparticles for Hepatocellular siRNA Delivery

Kathryn A. Whitehead et al.

ACS NANO (2012)

Article Chemistry, Multidisciplinary

Maximizing the Potency of siRNA Lipid Nanoparticles for Hepatic Gene Silencing In Vivo

Muthusamy Jayaraman et al.

ANGEWANDTE CHEMIE-INTERNATIONAL EDITION (2012)

Article Medicine, Research & Experimental

siRNA-optimized Modifications for Enhanced In Vivo Activity

Denise M. Kenski et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2012)

Review Biochemistry & Molecular Biology

Inhibiting the C5-C5a receptor axis

Trent M. Woodruff et al.

MOLECULAR IMMUNOLOGY (2011)

Article Biotechnology & Applied Microbiology

Elimination Pathways of Systemically Delivered siRNA

Yuanyu Huang et al.

MOLECULAR THERAPY (2011)

Article Biotechnology & Applied Microbiology

Rational design of cationic lipids for siRNA delivery

Sean C. Semple et al.

NATURE BIOTECHNOLOGY (2010)

Article Multidisciplinary Sciences

A randomized, double-blind, placebo-controlled study of an RNAi-based therapy directed against respiratory syncytial virus

John DeVincenzo et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2010)

Review Biochemistry & Molecular Biology

Identifying druggable disease-modifying gene products

Scott J. Dixon et al.

CURRENT OPINION IN CHEMICAL BIOLOGY (2009)

Article Multidisciplinary Sciences

Small interfering RNA-induced TLR3 activation inhibits blood and lymphatic vessel growth

Won Gil Cho et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2009)

Article Ophthalmology

COMBINATION THERAPY USING THE SMALL INTERFERING RNA BEVASIRANIB

Lawrence Singerman

RETINA-THE JOURNAL OF RETINAL AND VITREOUS DISEASES (2009)

Review Biochemistry & Molecular Biology

Problems associated with reporter assays in RNAi studies

Guihua Sun et al.

RNA BIOLOGY (2009)

Article Biochemistry & Molecular Biology

Trivalent, Gal/GalNAc-containing ligands designed for the asialoglycoprotein receptor

Oleg Khorev et al.

BIOORGANIC & MEDICINAL CHEMISTRY (2008)

Review Pharmacology & Pharmacy

Chemically modified siRNA: tools and applications

Jonathan K. Watts et al.

DRUG DISCOVERY TODAY (2008)

Article Multidisciplinary Sciences

Sequence- and target-independent angiogenesis suppression by siRNA via TLR3

Mark E. Kleinman et al.

NATURE (2008)

Article Biotechnology & Applied Microbiology

The impact of target site accessibility on the design of effective siRNAs

Hakim Tafer et al.

NATURE BIOTECHNOLOGY (2008)

Article Biotechnology & Applied Microbiology

A combinatorial library of lipid-like materials for delivery of RNAi therapeutics

Akin Akinc et al.

NATURE BIOTECHNOLOGY (2008)

Article Biochemistry & Molecular Biology

OligoWalk: an online siRNA design tool utilizing hybridization thermodynamics

Zhi John Lu et al.

NUCLEIC ACIDS RESEARCH (2008)

Article Biochemistry & Molecular Biology

Efficient siRNA selection using hybridization thermodynamics

Zhi John Lu et al.

NUCLEIC ACIDS RESEARCH (2008)

Article Biotechnology & Applied Microbiology

Discovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria

Russell P. Rother et al.

NATURE BIOTECHNOLOGY (2007)

Article Biochemistry & Molecular Biology

Effect of target secondary structure on RNAi efficiency

Yu Shao et al.

Article Biochemical Research Methods

A protocol for designing siRNAs with high functionality and specificity

Amanda Birmingham et al.

NATURE PROTOCOLS (2007)

Article Biochemistry & Molecular Biology

Controlling activation of the RNA-dependent protein kinase by siRNAs using site-specific chemical modification

Sujiet Puthenveetil et al.

NUCLEIC ACIDS RESEARCH (2006)

Article Biochemistry & Molecular Biology

Off-target effects by siRNA can induce toxic phenotype

Yuriy Fedorov et al.

Article Biochemistry & Molecular Biology

Improvements in siRNA properties mediated by 2 '-deoxy-2 '-fluoro-beta-D-arabinonucleic acid (FANA)

T Dowler et al.

NUCLEIC ACIDS RESEARCH (2006)

Review Biotechnology & Applied Microbiology

Activation of the mammalian immune system by siRNAs

JT Marques et al.

NATURE BIOTECHNOLOGY (2005)

Article Biochemistry & Molecular Biology

Using siRNA in prophylactic and therapeutic regimens against SARS coronavirus in rhesus macaque

BJ Li et al.

NATURE MEDICINE (2005)

Article Biochemistry & Molecular Biology

Sequence characteristics of functional siRNAs

B Jagla et al.

Article Biochemistry & Molecular Biology

Purified Argonaute2 and an siRNA form recombinant human RISC

FV Rivas et al.

NATURE STRUCTURAL & MOLECULAR BIOLOGY (2005)

Article Biochemistry & Molecular Biology

Algorithm for selection of functional siRNA sequences

M Amarzguioui et al.

BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS (2004)

Article Biotechnology & Applied Microbiology

Rational siRNA design for RNA interference

A Reynolds et al.

NATURE BIOTECHNOLOGY (2004)

Article Biochemistry & Molecular Biology

Sfold web server for statistical folding and rational design of nucleic acids

Y Ding et al.

NUCLEIC ACIDS RESEARCH (2004)

Article Multidisciplinary Sciences

Protection against lethal influenza virus challenge by RNA interference in vivo

SM Tompkins et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2004)

Article Biochemistry & Molecular Biology

Functional siRNAs and rniRNAs exhibit strand bias

A Khvorova et al.

Article Biotechnology & Applied Microbiology

Expression of small interfering RNAs targeted against HIV-1 rev transcripts in human cells

NS Lee et al.

NATURE BIOTECHNOLOGY (2002)

Article Multidisciplinary Sciences

RNA interference by expression of short-interfering RNAs and hairpin RNAs in mammalian cells

JY Yu et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2002)

Article Biochemistry & Molecular Biology

Functional anatomy of siRNAs for mediating efficient RNAi in Drosophila melanogaster embryo lysate

SM Elbashir et al.

EMBO JOURNAL (2001)

Article Multidisciplinary Sciences

Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells

SM Elbashir et al.

NATURE (2001)

Review Multidisciplinary Sciences

Initial sequencing and analysis of the human genome

ES Lander et al.

NATURE (2001)