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。siRNA therapeutics: a clinical reality
Phei Er Saw et al.
SCIENCE CHINA-LIFE SCIENCES (2020)
Synthesis, Derivatization and Photochemical Control of ortho-Functionalized Tetrachlorinated Azobenzene-Modified siRNAs
Matthew L. Hammill et al.
CHEMBIOCHEM (2020)
Effects of Chemical Modifications on siRNA Strand Selection in Mammalian Cells
Andrew J. Varley et al.
NUCLEIC ACID THERAPEUTICS (2020)
2′-O-Methyl at 20-mer Guide Strand 3′ Termini May Negatively Affect Target Silencing Activity of Fully Chemically Modified siRNA
Sarah M. Davis et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2020)
Chimeric siRNAs with chemically modified pentofuranose and hexopyranose nucleotides: altritol-nucleotide (ANA) containing GalNAc-siRNA conjugates: in vitro and in vivo RNAi activity and resistance to 5′-exonuclease
Pawan Kumar et al.
NUCLEIC ACIDS RESEARCH (2020)
Effect of 2′-5′/3′-5′ phosphodiester linkage heterogeneity on RNA interference
Maryam Habibian et al.
NUCLEIC ACIDS RESEARCH (2020)
Synthesis and Evaluation of Neutral Phosphate Triester Backbone-Modified siRNAs
Kouta Tsubaki et al.
ACS MEDICINAL CHEMISTRY LETTERS (2020)
Investigating the pharmacodynamic durability o f GalNAc-siRNA conjugates
Christopher R. Brown et al.
NUCLEIC ACIDS RESEARCH (2020)
Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects
Hongyi Li et al.
SIGNAL TRANSDUCTION AND TARGETED THERAPY (2020)
Strategies, design, and chemistry in siRNA delivery systems
Yizhou Dong et al.
ADVANCED DRUG DELIVERY REVIEWS (2019)
Current Aspects of siRNA Bioconjugate for In Vitro and In Vivo Delivery
Wanyi Tai
MOLECULES (2019)
Lipid Nanoparticle Technology for Clinical Translation of siRNA Therapeutics
Jayesh A. Kulkarni et al.
ACCOUNTS OF CHEMICAL RESEARCH (2019)
A systematic study on the influence of thermodynamic asymmetry of 5′-ends of siRNA duplexes in relation to their silencing potency
Jolanta Lisowiec-Wachnicka et al.
SCIENTIFIC REPORTS (2019)
5′-Morpholino modification of the sense strand of an siRNA makes it a more effective passenger
Pawan Kumar et al.
CHEMICAL COMMUNICATIONS (2019)
Targeted Delivery of an siRNA/PNA Hybrid Nanocomplex Reverses Carbon Tetrachloride-Induced Liver Fibrosis
Akshay Jain et al.
ADVANCED THERAPEUTICS (2019)
4′-Guanidinium-modified siRNA: a molecular tool to control RNAi activity through RISC priming and selective antisense strand loading
Ganesh N. Nawale et al.
CHEMICAL COMMUNICATIONS (2019)
A Single Amide Linkage in the Passenger Strand Suppresses Its Activity and Enhances Guide Strand Targeting of siRNAs
Travis Hardcastle et al.
ACS CHEMICAL BIOLOGY (2018)
Cholesterol-Modified Caged siRNAs for Photoregulating Exogenous and Endogenous Gene Expression
Jiali Yang et al.
BIOCONJUGATE CHEMISTRY (2018)
siRNA Modified with 2-Deoxy-2-C-methylpyrimidine Nucleosides
Maria Dellafiore et al.
CHEMBIOCHEM (2018)
Locked nucleic acid: modality, diversity, and drug discovery
Peter H. Hagedorn et al.
DRUG DISCOVERY TODAY (2018)
Advanced siRNA Designs Further Improve In Vivo Performance of GaINAc-siRNA Conjugates
Donald J. Foster et al.
MOLECULAR THERAPY (2018)
Comparison of partially and fully chemically-modified siRNA in conjugate-mediated delivery in vivo
Matthew R. Hassler et al.
NUCLEIC ACIDS RESEARCH (2018)
Selection of GalNAc-conjugated siRNAs with limited off-target-driven rat hepatotoxicity
Maja M. Janas et al.
NATURE COMMUNICATIONS (2018)
GalNAc-siRNA Conjugates: Leading the Way for Delivery of RNAi Therapeutics
Aaron D. Springer et al.
NUCLEIC ACID THERAPEUTICS (2018)
Effective carrier-free gene-silencing activity of cholesterol-modified siRNAs
Lidya Salim et al.
RSC ADVANCES (2018)
Circular siRNAs for Reducing Off-Target Effects and Enhancing Long-Term Gene Silencing in Cells and Mice
Liangliang Zhang et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2018)
Stability and evaluation of siRNAs labeled at the sense strand with a 3 '-azobenzene unit
Matthew L. Hammill et al.
BIOORGANIC & MEDICINAL CHEMISTRY LETTERS (2018)
Noncovalent Attachment of Chemical Moieties to siRNAs Using Peptide Nucleic Acid as a Complementary Linker
Wei Jin et al.
ACS APPLIED BIO MATERIALS (2018)
siRNA carrying an (E)-vinylphosphonate moiety at the 5′ end of the guide strand augments gene silencing by enhanced binding to human Argonaute-2
Elad Elkayam et al.
NUCLEIC ACIDS RESEARCH (2017)
Chirality Dependent Potency Enhancement and Structural Impact of Glycol Nucleic Acid Modification on siRNA
Mark K. Schlegel et al.
JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2017)
The chemical evolution of oligonucleotide therapies of clinical utility
Anastasia Khvorova et al.
NATURE BIOTECHNOLOGY (2017)
Overcoming cellular barriers for RNA therapeutics
Steven F. Dowdy
NATURE BIOTECHNOLOGY (2017)
A Highly Durable RNAi Therapeutic Inhibitor of PCSK9
Kevin Fitzgerald et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
Inclisiran in Patients at High Cardiovascular Risk with Elevated LDL Cholesterol
Kausik K. Ray et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
Amide linkages mimic phosphates in RNA interactions with proteins and are well tolerated in the guide strand of short interfering RNAs
Daniel Mutisya et al.
NUCLEIC ACIDS RESEARCH (2017)
5′-Vinylphosphonate improves tissue accumulation and efficacy of conjugated siRNAs in vivo
Reka A. Haraszti et al.
NUCLEIC ACIDS RESEARCH (2017)
Site-Specific Modification Using the 2′-Methoxyethyl Group Improves the Specificity and Activity of siRNAs
Xinyun Song et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2017)
Cholesterol-Containing Nuclease-Resistant siRNA Accumulates in Tumors in a Carrier-free Mode and Silences MDR1 Gene
Ivan V. Chernikov et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2017)
siRNAzos: A New Class of Azobenzene-Containing siRNAs that Can Photochemically Regulate Gene Expression
Matthew L. Hammill et al.
CHEMISTRYSELECT (2017)
Chemical Modification of the siRNA Seed Region Suppresses Off Target Effects by Steric Hindrance to Base-Pairing with Targets
Hanna Iribe et al.
ACS OMEGA (2017)
Synergistic effect of phosphorothioate, 5′-vinylphosphonate and GalNAc modifications for enhancing activity of synthetic siRNA
Thazha P. Prakash et al.
BIOORGANIC & MEDICINAL CHEMISTRY LETTERS (2016)
9 5′-(E)-Vinylphosphonate: A Stable Phosphate Mimic Can Improve the RNAi Activity of siRNA-GalNAc Conjugates
Rubina Parmar et al.
CHEMBIOCHEM (2016)
siRNA and RNAi optimization
Adele Alagia et al.
WILEY INTERDISCIPLINARY REVIEWS-RNA (2016)
Visualizing lipid-formulated siRNA release from endosomes and target gene knockdown
Anders Wittrup et al.
NATURE BIOTECHNOLOGY (2015)
Identification of metabolically stable 5′- phosphate analogs that support single-stranded siRNA activity
Thazha P. Prakash et al.
NUCLEIC ACIDS RESEARCH (2015)
Terminus-free siRNA prepared by photo-crosslinking activated via slicing by Ago2
Yukiko Kamiya et al.
BIOMATERIALS SCIENCE (2015)
Hydrophobically Modified siRNAs Silence Huntingtin mRNA in Primary Neurons and Mouse Brain
Julia F. Alterman et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2015)
siRNA Versus miRNA as Therapeutics for Gene Silencing
Jenny K. W. Lam et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2015)
Endogenous siRNAs: regulators of internal affairs
Monica J. Piatek et al.
BIOCHEMICAL SOCIETY TRANSACTIONS (2014)
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)
Single modification at position 14 of siRNA strand abolishes its gene-silencing activity by decreasing both RISC loading and target degradation
Jie Zheng et al.
FASEB JOURNAL (2013)
Multiple sensors ensure guide strand selection in human RNAi pathways
Cameron L. Noland et al.
RNA (2013)
Single-Stranded RNAs Use RNAi to Potently and Allele-Selectively Inhibit Mutant Huntingtin Expression
Dongbo Yu et al.
CELL (2012)
Single-Stranded siRNAs Activate RNAi in Animals
Walt F. Lima et al.
CELL (2012)
Modification of the siRNA Passenger Strand by 5-Nitroindole Dramatically Reduces its Off-Target Effects
Junbin Zhang et al.
CHEMBIOCHEM (2012)
Asymmetric siRNA: New Strategy to Improve Specificity and Reduce Off-Target Gene Expression
Zhongping Yuan et al.
HUMAN GENE THERAPY (2012)
Lipid-Conjugated 27-Nucleotide Double-Stranded RNAs with Dicer-Substrate Potency Enhance RNAi-Mediated Gene Silencing
Takanori Kubo et al.
MOLECULAR PHARMACEUTICS (2012)
The Crystal Structure of Human Argonaute2
Nicole T. Schirle et al.
SCIENCE (2012)
Defined Folate-PEG-siRNA Conjugates for Receptor-specific Gene Silencing
Christian Dohmen et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2012)
Amides as Excellent Mimics of Phosphate Linkages in RNA
Chelliah Selvam et al.
ANGEWANDTE CHEMIE-INTERNATIONAL EDITION (2011)
Synthesis, Structure, and Biological Activity of Dumbbell-Shaped Nanocircular RNAs for RNA Interference
Naoko Abe et al.
BIOCONJUGATE CHEMISTRY (2011)
Modeling RNA interference in mammalian cells
Giulia Cuccato et al.
BMC SYSTEMS BIOLOGY (2011)
Nucleoside Optimization for RNAi: A High-Throughput Platform
Gabor Butora et al.
JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2011)
Modified siRNA Structure With a Single Nucleotide Bulge Overcomes Conventional siRNA-mediated Off-target Silencing
Pooja Dua et al.
MOLECULAR THERAPY (2011)
Elimination Pathways of Systemically Delivered siRNA
Yuanyu Huang et al.
MOLECULAR THERAPY (2011)
Unexpected origins of the enhanced pairing affinity of 2′-fluoro-modified RNA
Pradeep S. Pallan et al.
NUCLEIC ACIDS RESEARCH (2011)
Improved specificity of gene silencing by siRNAs containing unlocked nucleobase analogs
Narendra Vaish et al.
NUCLEIC ACIDS RESEARCH (2011)
Application of small interfering RNAs modified by unlocked nucleic acid (UNA) to inhibit the heart-pathogenic coxsackievirus B3
Denise Werk et al.
FEBS LETTERS (2010)
Configuration of the 5′-Methyl Group Modulates the Biophysical and Biological Properties of Locked Nucleic Acid (LNA) Oligonucleotides
Punit P. Seth et al.
JOURNAL OF MEDICINAL CHEMISTRY (2010)
Utilization of unlocked nucleic acid (UNA) to enhance siRNA performance in vitro and in vivo
Maria B. Laursen et al.
MOLECULAR BIOSYSTEMS (2010)
Diverse Endonucleolytic Cleavage Sites in the Mammalian Transcriptome Depend upon MicroRNAs, Drosha, and Additional Nucleases
Fedor V. Karginov et al.
MOLECULAR CELL (2010)
Structural basis for 5′-nucleotide base-specific recognition of guide RNA by human AGO2
Filipp Frank et al.
NATURE (2010)
A Dicer-independent miRNA biogenesis pathway that requires Ago catalysis
Sihem Cheloufi et al.
NATURE (2010)
Analysis of acyclic nucleoside modifications in siRNAs finds sensitivity at position 1 that is restored by 5'-terminal phosphorylation both in vitro and in vivo
Denise M. Kenski et al.
NUCLEIC ACIDS RESEARCH (2010)
A screen of chemical modifications identifies position-specific modification by UNA to most potently reduce siRNA off-target effects
Jesper B. Bramsen et al.
NUCLEIC ACIDS RESEARCH (2010)
A Novel miRNA Processing Pathway Independent of Dicer Requires Argonaute2 Catalytic Activity
Daniel Cifuentes et al.
SCIENCE (2010)
Asymmetric Shorter-duplex siRNA Structures Trigger Efficient Gene Silencing With Reduced Nonspecific Effects
Chan Il Chang et al.
MOLECULAR THERAPY (2009)
Both Strands of siRNA Have Potential to Guide Posttranscriptional Gene Silencing in Mammalian Cells
Jun-Xia Wei et al.
PLOS ONE (2009)
Efficient in vivo delivery of siRNA to the liver by conjugation of α-tocopherol
Kazutaka Nishina et al.
MOLECULAR THERAPY (2008)
Effect of asymmetric terminal structures of short RNA duplexes on the RNA interference activity and strand selection
Masayuki Sano et al.
NUCLEIC ACIDS RESEARCH (2008)
Experimental validation of the importance of seed complement frequency to siRNA specificity
Emily M. Anderson et al.
RNA (2008)
Strand-specific 5′-O-methylation of siRNA duplexes controls guide strand selection and targeting specificity
Po Yu Chen et al.
RNA (2008)
Impact of tumor-specific targeting on the biodistribution and efficacy of siRNA nanoparticles measured by multimodality in vivo imaging
Derek W. Bartlett et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2007)
Lung delivery studies using siRNA conjugated to TAT(48-60) and penetratin reveal peptide induced reduction in gene expression and induction of innate immunity
Sterghios Athanasios Moschos et al.
BIOCONJUGATE CHEMISTRY (2007)
hClp1 - A novel kinase revitalizes RNA metabolism
Stefan Weitzer et al.
CELL CYCLE (2007)
Improved silencing properties using small internally segmented interfering RNAs
Jesper B. Bramsen et al.
NUCLEIC ACIDS RESEARCH (2007)
The human RNA kinase hClp1 is active on 3′ transfer RNA exons and short interfering RNAs
Stefan Weitzer et al.
NATURE (2007)
Evaluation of locked nucleic acid-modified small interfering RNA in vitro and in vivo
Olaf R. Mook et al.
MOLECULAR CANCER THERAPEUTICS (2007)
Solid-phase synthesis of modified RNAs containing amide-linked oligoribonucleosides at their 3 '-end and their application to siRNA
Reiko Iwase et al.
NUCLEOSIDES NUCLEOTIDES & NUCLEIC ACIDS (2007)
Tolerance of RNA interference toward modifications of the 5 ' antisense phosphate of small interfering RNA
Samit Shah et al.
OLIGONUCLEOTIDES (2007)
Cell type-specific delivery of siRNAs with aptamer-siRNA chimeras
James O. McNamara et al.
NATURE BIOTECHNOLOGY (2006)
Widespread siRNA off-target'' transcript silencing mediated by seed region sequence complementarity
Aimee L. Jackson et al.
RNA (2006)
3′ UTR seed matches, but not overall identity, are associated with RNAi off-targets
A Birmingham et al.
NATURE METHODS (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)
siRNA-mediated off-target gene silencing triggered by a 7 nt complementation
XY Lin et al.
NUCLEIC ACIDS RESEARCH (2005)
Locked nucleic acid (LNA) mediated improvements in siRNA stability and functionality
J Elmen et al.
NUCLEIC ACIDS RESEARCH (2005)
Argonaute2 is the catalytic engine of mammalian RNAi
JD Liu et al.
SCIENCE (2004)
Steroid and lipid conjugates of siRNAs to enhance cellular uptake and gene silencing in liver cells
C Lorenz et al.
BIOORGANIC & MEDICINAL CHEMISTRY LETTERS (2004)
Functional siRNAs and rniRNAs exhibit strand bias
A Khvorova et al.
CELL (2003)
Asymmetry in the assembly of the RNAi enzyme complex
DS Schwarz et al.
CELL (2003)
siRNA function in RNAi: A chemical modification analysis
YL Chiu et al.
RNA (2003)
RNA interference in mammalian cells by chemically-modified RNA
DA Braasch et al.
BIOCHEMISTRY (2003)
Expression profiling reveals off-target gene regulation by RNAi
AL Jackson et al.
NATURE BIOTECHNOLOGY (2003)
Single-stranded antisense siRNAs guide target RNA cleavage in RNAi
J Martinez et al.
CELL (2002)
ATP requirements and small interfering RNA structure in the RNA interference pathway
A Nykänen et al.
CELL (2001)