4.7 Review

siRNA therapeutics: a clinical reality

Related references

Note: Only part of the references are listed.
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

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)

Article Chemistry, Multidisciplinary

Tumor Microenvironment-Responsive Multistaged Nanoplatform for Systemic RNAi and Cancer Therapy

Xiaoding Xu et al.

NANO LETTERS (2017)

Article Nanoscience & Nanotechnology

A drug-delivery strategy for overcoming drug resistance in breast cancer through targeting of oncofetal fibronectin

Phei Er Saw et al.

NANOMEDICINE-NANOTECHNOLOGY BIOLOGY AND MEDICINE (2017)

Article Biochemistry & Molecular Biology

Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc-siRNA conjugates

Jayaprakash K. Nair et al.

NUCLEIC ACIDS RESEARCH (2017)

Review Oncology

Drugging the 'undruggable' cancer targets

Chi V. Dang et al.

NATURE REVIEWS CANCER (2017)

Article Chemistry, Multidisciplinary

Ultra-pH-Responsive and Tumor-Penetrating Nanoplatform for Targeted siRNA Delivery with Robust Anti-Cancer Efficacy

Xiaoding Xu et al.

ANGEWANDTE CHEMIE-INTERNATIONAL EDITION (2016)

Review Nanoscience & Nanotechnology

Lipid nanoparticles for targeted siRNA delivery - going from bench to bedside

Timofei S. Zatsepin et al.

INTERNATIONAL JOURNAL OF NANOMEDICINE (2016)

Review Clinical Neurology

Red-flag symptom clusters in transthyretin familial amyloid polyneuropathy

Isabel Conceicao et al.

JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM (2016)

Article Medicine, General & Internal

Experimental Treatment of Ebola Virus Disease with TKM-130803: A Single-Arm Phase 2 Clinical Trial

Jake Dunning et al.

PLOS MEDICINE (2016)

Review Pharmacology & Pharmacy

Preclinical and clinical development of siRNA-based therapeutics

Gulnihal Ozcan et al.

ADVANCED DRUG DELIVERY REVIEWS (2015)

Review Medicine, General & Internal

Evolving landscape in the management of transthyretin amyloidosis

Philip N. Hawkins et al.

ANNALS OF MEDICINE (2015)

Editorial Material Medicine, General & Internal

Harnessing RNA Interference for Therapy The Silent Treatment

Judy Lieberman et al.

JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION (2015)

Review Biotechnology & Applied Microbiology

Clinical experiences with systemically administered siRNA-based therapeutics in cancer

Jonathan E. Zuckerman et al.

NATURE REVIEWS DRUG DISCOVERY (2015)

Review Genetics & Heredity

Knocking down disease: a progress report on siRNA therapeutics

Anders Wittrup et al.

NATURE REVIEWS GENETICS (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 Pharmacology & Pharmacy

Cationic cell-penetrating peptides as vehicles for siRNA delivery

Jagadish Beloor et al.

THERAPEUTIC DELIVERY (2015)

Article Engineering, Biomedical

Multifunctional, self-assembling anionic peptide-lipid nanocomplexes for targeted siRNA delivery

Aristides D. Tagalakis et al.

BIOMATERIALS (2014)

Article Chemistry, Multidisciplinary

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)

Article Chemistry, Multidisciplinary

Adsorption and Release of siRNA from Porous Silica

Jeremy L. Steinbacher et al.

LANGMUIR (2014)

Meeting Abstract Cardiac & Cardiovascular Systems

Phase I First-in-Humans Trial of ALN-TTRsc, a Novel RNA Interference Therapeutic for the Treatment of Familial Amyloidotic Cardiomyopathy (FAC)

Tracy Zimmermann et al.

JOURNAL OF CARDIAC FAILURE (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)

Review Genetics & Heredity

Guideline of transthyretin-related hereditary amyloidosis for clinicians

Yukio Ando et al.

ORPHANET JOURNAL OF RARE DISEASES (2013)

Review Biochemistry & Molecular Biology

RNA-Based Therapeutics: Current Progress and Future Prospects

John C. Burnett et al.

CHEMISTRY & BIOLOGY (2012)

Article Pharmacology & Pharmacy

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

Dirk Strumberg et al.

INTERNATIONAL JOURNAL OF CLINICAL PHARMACOLOGY AND THERAPEUTICS (2012)

Article Medicine, Research & Experimental

siRNA-optimized Modifications for Enhanced In Vivo Activity

Denise M. Kenski et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2012)

Article Critical Care Medicine

RNA Interference Therapy in Lung Transplant Patients Infected with Respiratory Syncytial Virus

Martin R. Zamora et al.

AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE (2011)

Review Biochemical Research Methods

Current progress of siRNA/shRNA therapeutics in clinical trials

John C. Burnett et al.

BIOTECHNOLOGY JOURNAL (2011)

Review Biotechnology & Applied Microbiology

Nanocarriers: a tool to overcome biological barriers in siRNA delivery

Avnesh Kumari et al.

EXPERT OPINION ON BIOLOGICAL THERAPY (2011)

Article Oncology

Promise and Challenge of RNA Interference-Based Therapy for Cancer

Fabio Petrocca et al.

JOURNAL OF CLINICAL ONCOLOGY (2011)

Article Biochemistry & Molecular Biology

siRNA-induced liver ApoB knockdown lowers serum LDL-cholesterol in a mouse model with human-like serum lipids

Marija Tadin-Strapps et al.

JOURNAL OF LIPID RESEARCH (2011)

Review Cell Biology

The ups and downs of Myc biology

Laura Soucek et al.

CURRENT OPINION IN GENETICS & DEVELOPMENT (2010)

Article Hematology

Small interfering RNA against BCR-ABL transcripts sensitize mutated T315I cells to nilotinib

Michael Koldehoff et al.

HAEMATOLOGICA-THE HEMATOLOGY JOURNAL (2010)

Article Biotechnology & Applied Microbiology

First-in-human Mutation-targeted siRNA Phase Ib Trial of an Inherited Skin Disorder

Sancy A. Leachman et al.

MOLECULAR THERAPY (2010)

Review Oncology

Delivering nanomedicine to solid tumors

Rakesh K. Jain et al.

NATURE REVIEWS CLINICAL ONCOLOGY (2010)

Review Biotechnology & Applied Microbiology

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

Aimee L. Jackson et al.

NATURE REVIEWS DRUG DISCOVERY (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 Biochemical Research Methods

siRNA Conjugate Delivery Systems

Ji Hoon Jeong et al.

BIOCONJUGATE CHEMISTRY (2009)

Article Chemistry, Multidisciplinary

Layer-by-Layer Assembled Gold Nanoparticles for siRNA Delivery

Asmaa Elbakry et al.

NANO LETTERS (2009)

Review Biotechnology & Applied Microbiology

Knocking down barriers: advances in siRNA delivery

Kathryn A. Whitehead et al.

NATURE REVIEWS DRUG DISCOVERY (2009)

Article Biochemistry & Molecular Biology

RNA major groove modifications improve siRNA stability and biological activity

Montserrat Terrazas et al.

NUCLEIC ACIDS RESEARCH (2009)

Review Biochemistry & Molecular Biology

siRNA and Innate Immunity

Marjorie Robbins et al.

OLIGONUCLEOTIDES (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)

Article Ophthalmology

COMPARISON OF INTRAVITREAL BEVACIZUMAB FOLLOWED BY RANIBIZUMAB FOR THE TREATMENT OF NEOVASCULAR AGE-RELATED MACULAR DEGENERATION

Kimberly E. Stepien et al.

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

Article Biotechnology & Applied Microbiology

Misinterpreting the Therapeutic Effects of Small Interfering RNA Caused by Immune Stimulation

Marjorie Robbins et al.

HUMAN GENE THERAPY (2008)

Review Biotechnology & Applied Microbiology

Delivery vehicles for small interfering RNA in vivo

Antonin R. De Fougerolles

HUMAN GENE THERAPY (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

Resolution of liver cirrhosis using vitamin A-coupled liposomes to deliver siRNA against a collagen-specific chaperone

Yasushi Sato et al.

NATURE BIOTECHNOLOGY (2008)

Review Medicine, Research & Experimental

Nonviral delivery of synthetic siRNAs in vivo

Saghir Akhtar et al.

JOURNAL OF CLINICAL INVESTIGATION (2007)

Review Biotechnology & Applied Microbiology

Interfering with disease: a progress report on siRNA-based therapeutics

Antonin de Fougerolles et al.

NATURE REVIEWS DRUG DISCOVERY (2007)

Review Biochemistry & Molecular Biology

RNAi therapeutics: a potential new class of pharmaceutical drugs

David Bumcrot et al.

NATURE CHEMICAL BIOLOGY (2006)

Review Biochemistry & Molecular Biology

A brief history of RNAi: the silence of the genes

George L. Sen et al.

FASEB JOURNAL (2006)

Article Multidisciplinary Sciences

RNAi-mediated gene silencing in non-human primates

TS Zimmermann et al.

NATURE (2006)

Article Biochemistry & Molecular Biology

Chemical modification of siRNAs to improve serum stability without loss of efficacy

S Choung et al.

BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS (2006)

Review Biotechnology & Applied Microbiology

Progress towards in vivo use of siRNAs

MA Behlke

MOLECULAR THERAPY (2006)

Article Biochemical Research Methods

3′ UTR seed matches, but not overall identity, are associated with RNAi off-targets

A Birmingham et al.

NATURE METHODS (2006)

Article Biotechnology & Applied Microbiology

Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs

DV Morrissey et al.

NATURE BIOTECHNOLOGY (2005)

Article Biotechnology & Applied Microbiology

Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors

EW Song et al.

NATURE BIOTECHNOLOGY (2005)

Article Gastroenterology & Hepatology

Activity of stabilized short interfering RNA in a mouse model of hepatitis B virus replication

DV Morrissey et al.

HEPATOLOGY (2005)

Article Biotechnology & Applied Microbiology

Sequence-dependent stimulation of the mammalian innate immune response by synthetic siRNA

AD Judge et al.

NATURE BIOTECHNOLOGY (2005)

Article Chemistry, Multidisciplinary

A scalable, extrusion-free method for efficient liposomal encapsulation of plasmid DNA

LB Jeffs et al.

PHARMACEUTICAL RESEARCH (2005)

Review Medicine, Research & Experimental

The silent revolution: RNA interference as basic biology, research tool, and therapeutic

DM Dykxhoorn et al.

ANNUAL REVIEW OF MEDICINE (2005)

Review Biochemistry & Molecular Biology

siRNA therapeutics: Big potential from small RNAs

RCC Ryther et al.

GENE THERAPY (2005)

Article Biochemistry & Molecular Biology

In vivo activity of nuclease-resistant siRNAs

JM Layzer et al.

Article Multidisciplinary Sciences

Small interfering RNA targeting Fas protects mice against renal ischemia-reperfusion injury

P Hamar et al.

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

Article Cell Biology

Activation of the interferon system by short-interfering RNAs

CA Sledz et al.

NATURE CELL BIOLOGY (2003)

Review Biochemistry & Molecular Biology

Interfering with disease: opportunities and roadblocks to harnessing RNA interference

J Lieberman et al.

TRENDS IN MOLECULAR MEDICINE (2003)

Article Biotechnology & Applied Microbiology

Expression profiling reveals off-target gene regulation by RNAi

AL Jackson et al.

NATURE BIOTECHNOLOGY (2003)

Article Biochemistry & Molecular Biology

RNA interference targeting Fas protects mice from fulminant hepatitis

EW Song et al.

NATURE MEDICINE (2003)

Article Multidisciplinary Sciences

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

SM Elbashir et al.

NATURE (2001)