Overcoming Barriers for siRNA Therapeutics: From Bench to Bedside

Givosiran: First Approval

(2020) Lesley J. Scott   DRUGS

Nucleic acid-based drug delivery strategies

(2020) Xuyu Tan et al.   JOURNAL OF CONTROLLED RELEASE

Lipid and Polymer-Based Nanoparticle siRNA Delivery Systems for Cancer Therapy

(2020) Francesco Mainini et al.   MOLECULES

siRNA based drug design, quality, delivery and clinical translation

(2020) Md Abdus Subhan et al.   Nanomedicine-Nanotechnology Biology and Medicine

The current state and future directions of RNAi-based therapeutics

(2019) Ryan L. Setten et al.   NATURE REVIEWS DRUG DISCOVERY

Double-Stranded RNA Sensors and Modulators in Innate Immunity

(2019) Sun Hur   Annual Review of Immunology

Binary Targeting of siRNA to Hematologic Cancer Cells In Vivo Using Layer‐by‐Layer Nanoparticles

(2019) Ki Young Choi et al.   ADVANCED FUNCTIONAL MATERIALS

RNAi therapeutic and its innovative biotechnological evolution

(2019) Yuhua Weng et al.   BIOTECHNOLOGY ADVANCES

Oral delivery of non-viral nucleic acid-based therapeutics - do we have the guts for this?

(2019) Caitriona M. O'Driscoll et al.   EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES

Effectiveness of siRNA Delivery via Arginine-Rich PEI-based Polyplex in Metastatic and Doxorubicin Resistant Breast Cancer Cells

(2019) Shan Lu et al.   JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS

Current Aspects of siRNA Bioconjugate for In Vitro and In Vivo Delivery

(2019) Wanyi Tai   MOLECULES

N6-methyl adenosine in siRNA evades immune response without reducing RNAi activity

(2019) Akihiro Imaeda et al.   NUCLEOSIDES NUCLEOTIDES & NUCLEIC ACIDS

PKR activation by non-canonical ligands: a 5′-triphosphate requirement versus antisense contamination.

(2019) Sarah A Safran et al.   RNA

Peptide carriers to the rescue: Overcoming the barriers to siRNA delivery for cancer treatment

(2019) James C. Cummings et al.   Translational Research

Endosomal TLR-8 Senses microRNA-1294 Resulting in the Production of NFḱB Dependent Cytokines

(2019) Linda Pluta et al.   Frontiers in Immunology

Recent advances in siRNA delivery for cancer therapy using smart nanocarriers

(2018) Penghui Zhang et al.   DRUG DISCOVERY TODAY

siRNA delivery for treatment of degenerative diseases, new hopes and challenges

(2018) Forouhe Zahir-Jouzdani et al.   JOURNAL OF DRUG DELIVERY SCIENCE AND TECHNOLOGY

Selection of GalNAc-conjugated siRNAs with limited off-target-driven rat hepatotoxicity

(2018) Maja M. Janas et al.   Nature Communications

Exosome-based small RNA delivery: Progress and prospects

(2018) Mei Lu et al.   Asian Journal of Pharmaceutical Sciences

Systemic siRNA delivery to tumors by cell-penetrating α-helical polypeptide-based metastable nanoparticles

(2018) Yang Liu et al.   Nanoscale

Controlling endosomal escape using nanoparticle composition: current progress and future perspectives

(2018) Kristofer I Cupic et al.   Nanomedicine

Structural Analyses of Toll-like Receptor 7 Reveal Detailed RNA Sequence Specificity and Recognition Mechanism of Agonistic Ligands

(2018) Zhikuan Zhang et al.   Cell Reports

The Toll-like receptor 9 signalling pathway regulates MR1-mediated bacterial antigen presentation in B cells

(2017) Jianyun Liu et al.   IMMUNOLOGY

Enhancing the Therapeutic Delivery of Oligonucleotides by Chemical Modification and Nanoparticle Encapsulation

(2017) Yating Sun et al.   MOLECULES

Overcoming cellular barriers for RNA therapeutics

(2017) Steven F Dowdy   NATURE BIOTECHNOLOGY

Cholesterol-Containing Nuclease-Resistant siRNA Accumulates in Tumors in a Carrier-free Mode and Silences MDR1 Gene

(2017) Ivan V. Chernikov et al.   Molecular Therapy-Nucleic Acids

siRNA Delivery Strategies: A Comprehensive Review of Recent Developments

(2017) Katyayani Tatiparti et al.   Nanomaterials

RNA Interference-Induced Innate Immunity, Off-Target Effect, or Immune Adjuvant?

(2017) Zhongji Meng et al.   Frontiers in Immunology

Discriminating Self and Non-Self by RNA: Roles for RNA Structure, Misfolding, and Modification in Regulating the Innate Immune Sensor PKR

(2016) Chelsea M. Hull et al.   ACCOUNTS OF CHEMICAL RESEARCH

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

(2016) E Fakhr et al.   CANCER GENE THERAPY

Bioresponsive materials

(2016) Yue Lu et al.   Nature Reviews Materials

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

Clinical experiences with systemically administered siRNA-based therapeutics in cancer

(2015) Jonathan E. Zuckerman et al.   NATURE REVIEWS DRUG DISCOVERY

Chemically defined polyethylene glycol siRNA conjugates with enhanced gene silencing effect

(2014) Zuzana Gaziova et al.   BIOORGANIC & MEDICINAL CHEMISTRY

First-in-Human Phase I Study of the Liposomal RNA Interference Therapeutic Atu027 in Patients With Advanced Solid Tumors

(2014) Beate Schultheis et al.   JOURNAL OF CLINICAL ONCOLOGY

Development of RNAi technology for targeted therapy — A track of siRNA based agents to RNAi therapeutics

(2014) Yinjian Zhou et al.   JOURNAL OF CONTROLLED RELEASE

Identification of RNA Sequence Motifs Stimulating Sequence-Specific TLR8-Dependent Immune Responses

(2014) A. Forsbach et al.   JOURNAL OF IMMUNOLOGY

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

Correlating animal and human phase Ia/Ib clinical data with CALAA-01, a targeted, polymer-based nanoparticle containing siRNA

(2014) J. E. Zuckerman et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Nanoparticle-Mediated Systemic Delivery of siRNA for Treatment of Cancers and Viral Infections

(2014) Mohamed Shehata Draz et al.   Theranostics

A phase 1 study of the BCL2-targeted deoxyribonucleic acid inhibitor (DNAi) PNT2258 in patients with advanced solid tumors

(2013) Anthony W. Tolcher et al.   CANCER CHEMOTHERAPY AND PHARMACOLOGY

Exosomes are natural carriers of exogenous siRNA to human cells in vitro

(2013) Tatyana A Shtam et al.   Cell Communication and Signaling

siRNA delivery: from basics to therapeutic applications

(2013) Vladimir Torchilin   Frontiers in Bioscience-Landmark

Molecular basis for improved gene silencing by Dicer substrate interfering RNA compared with other siRNA variants

(2013) Nicholas M. Snead et al.   NUCLEIC ACIDS RESEARCH

First-in-Humans Trial of an RNA Interference Therapeutic Targeting VEGF and KSP in Cancer Patients with Liver Involvement

(2013) Josep Tabernero et al.   Cancer Discovery

Optimized siRNA-PEG Conjugates for Extended Blood Circulation and Reduced Urine Excretion in Mice

(2013) Frank Iversen et al.   Theranostics

5′ Unlocked Nucleic Acid Modification Improves siRNA Targeting

(2013) Nicholas M Snead et al.   Molecular Therapy-Nucleic Acids

Achieving efficient RNAi therapy: progress and challenges

(2013) Kun Gao et al.   Acta Pharmaceutica Sinica B

Safety profile of RNAi nanomedicines

(2012) Scott A. Barros et al.   ADVANCED DRUG DELIVERY REVIEWS

Biodegradable Nanoparticles of mPEG-PLGA-PLL Triblock Copolymers as Novel Non-Viral Vectors for Improving siRNA Delivery and Gene Silencing

(2012) Jing Du et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Clinical translation of RNAi-based treatments for respiratory diseases

(2012) Borja Ballarín-González et al.   Drug Delivery and Translational Research

Special delivery: targeted therapy with small RNAs

(2011) D Peer et al.   GENE THERAPY

Phase I clinical development of Atu027, a siRNA formulation targeting PKN3 in patients with advanced solid tumors

(2011) Dirk Strumberg et al.   INTERNATIONAL JOURNAL OF CLINICAL PHARMACOLOGY AND THERAPEUTICS

Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes

(2011) Lydia Alvarez-Erviti et al.   NATURE BIOTECHNOLOGY

SiRNA delivery with exosome nanoparticles

(2011) Jasper G van den Boorn et al.   NATURE BIOTECHNOLOGY

Delivery of siRNA Therapeutics: Barriers and Carriers

(2010) Jie Wang et al.   AAPS Journal

RNA interference of MBD1 in BxPC-3 human pancreatic cancer cells delivered by PLGA-poloxamer nanoparticles

(2010) Guopei Luo et al.   CANCER BIOLOGY & THERAPY

Targeted Gene Silencing Using RGD-Labeled Chitosan Nanoparticles

(2010) H. D. Han et al.   CLINICAL CANCER RESEARCH

Disulfide crosslinked stearoyl carrier peptides containing arginine and histidine enhance siRNA uptake and gene silencing

(2010) Ko Tanaka et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS

Breaking down the barriers: siRNA delivery and endosome escape

(2010) M. Dominska et al.   JOURNAL OF CELL SCIENCE

Endosomal escape pathways for delivery of biologicals

(2010) Amir K. Varkouhi et al.   JOURNAL OF CONTROLLED RELEASE

A dicer-independent miRNA biogenesis pathway that requires Ago catalysis

(2010) Sihem Cheloufi et al.   NATURE

Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles

(2010) Mark E. Davis et al.   NATURE

Recent advances in small interfering RNA sensing by the immune system

(2010) Mouldy Sioud   New Biotechnology

Directed evolution of gold nanoparticle delivery to cells

(2009) Na Li et al.   CHEMICAL COMMUNICATIONS

Cutaneous short-interfering RNA therapy

(2009) B Geusens et al.   Expert Opinion on Drug Delivery

A Nucleolin-Targeted Multimodal Nanoparticle Imaging Probe for Tracking Cancer Cells Using an Aptamer

(2009) D. W. Hwang et al.   JOURNAL OF NUCLEAR MEDICINE

The First Targeted Delivery of siRNA in Humans via a Self-Assembling, Cyclodextrin Polymer-Based Nanoparticle: From Concept to Clinic

(2009) Mark E. Davis   MOLECULAR PHARMACEUTICS

Systemic administration of optimized aptamer-siRNA chimeras promotes regression of PSMA-expressing tumors

(2009) Justin P Dassie et al.   NATURE BIOTECHNOLOGY

Knocking down barriers: advances in siRNA delivery

(2009) Kathryn A. Whitehead et al.   NATURE REVIEWS DRUG DISCOVERY

Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application

(2009) Aimee L. Jackson et al.   NATURE REVIEWS DRUG DISCOVERY

A large-scale chemical modification screen identifies design rules to generate siRNAs with high activity, high stability and low toxicity

(2009) Jesper B. Bramsen et al.   NUCLEIC ACIDS RESEARCH

siRNA and Innate Immunity

(2009) Marjorie Robbins et al.   OLIGONUCLEOTIDES

Therapeutic Applications of DNA and RNA Aptamers

(2009) Kristina W. Thiel et al.   OLIGONUCLEOTIDES

Selective Protection of Nuclease-Sensitive Sites in siRNA Prolongs Silencing Effect

(2009) Anton A. Volkov et al.   OLIGONUCLEOTIDES

Strategies for targeted nonviral delivery of siRNAs in vivo

(2009) Sang-Soo Kim et al.   TRENDS IN MOLECULAR MEDICINE

Cell-Specific Induction of Apoptosis by Rationally Designed Bivalent Aptamer-siRNA Transcripts Silencing Eukaryotic Elongation Factor 2

(2008) Ulrich Wullner et al.   CURRENT CANCER DRUG TARGETS

Overcoming the Innate Immune Response to Small Interfering RNA

(2008) Adam Judge et al.   HUMAN GENE THERAPY

RNA Interference in Mammalia Cells by RNA-3’-PNA Chimeras

(2008) Nicoletta Potenza et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Triggered release of siRNA from poly(ethylene glycol)-protected, pH-dependent liposomes

(2008) D AUGUSTE et al.   JOURNAL OF CONTROLLED RELEASE

Quantitative and Temporal Analysis of Gene Silencing in Tumor Cells Induced by Small Interfering RNA or Short Hairpin RNA Expressed from Plasmid Vectors

(2008) Yuki Takahashi et al.   JOURNAL OF PHARMACEUTICAL SCIENCES

Phase I Clinical Trial of Autologous Ascites-derived Exosomes Combined With GM-CSF for Colorectal Cancer

(2008) Shengming Dai et al.   MOLECULAR THERAPY

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

(2008) Mark E. Kleinman et al.   NATURE

Aptamer-based endocytosis of a lysosomal enzyme

(2008) C.-h. B. Chen et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA