4.7 Review

Silencing disease genes in the laboratory and the clinic

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。
Review Cardiac & Cardiovascular Systems

Therapeutic Advances in MicroRNA Targeting

Rusty L. Montgomery et al.

JOURNAL OF CARDIOVASCULAR PHARMACOLOGY (2011)

Review Oncology

miRNAs in human cancer

Thalia A. Farazi et al.

JOURNAL OF PATHOLOGY (2011)

News Item Biotechnology & Applied Microbiology

RNAi momentum fizzles as pharma shifts priorities

Charles Schmidt

NATURE BIOTECHNOLOGY (2011)

Article Medicine, General & Internal

Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

Nathalie M. Goemans et al.

NEW ENGLAND JOURNAL OF MEDICINE (2011)

Article Biochemistry & Molecular Biology

Mechanisms of single-stranded phosphorothioate modified antisense oligonucleotide accumulation in hepatocytes

Erich Koller et al.

NUCLEIC ACIDS RESEARCH (2011)

Article Biochemistry & Molecular Biology

Potent and systematic RNAi mediated silencing with single oligonucleotide compounds

Jennifer Lapierre et al.

Review Cell Biology

Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy

Aurelie Goyenvalle et al.

SKELETAL MUSCLE (2011)

Review Pharmacology & Pharmacy

Delivery of siRNA Therapeutics: Barriers and Carriers

Jie Wang et al.

AAPS JOURNAL (2010)

Review Pharmacology & Pharmacy

RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform

C. Frank Bennett et al.

ANNUAL REVIEW OF PHARMACOLOGY AND TOXICOLOGY (2010)

Review Biochemistry & Molecular Biology

Morpholinos and their peptide conjugates: Therapeutic promise and challenge for Duchenne muscular dystrophy

Hong M. Moulton et al.

BIOCHIMICA ET BIOPHYSICA ACTA-BIOMEMBRANES (2010)

Article Chemistry, Medicinal

Clinical status of duplex RNA

Jonathan K. Watts et al.

BIOORGANIC & MEDICINAL CHEMISTRY LETTERS (2010)

Article Cardiac & Cardiovascular Systems

Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers

Natee Jearawiriyapaisarn et al.

CARDIOVASCULAR RESEARCH (2010)

Article Oncology

Targeting the Cytoprotective Chaperone, Clusterin, for Treatment of Advanced Cancer

Amina Zoubeidi et al.

CLINICAL CANCER RESEARCH (2010)

Article Biotechnology & Applied Microbiology

Functional Rescue of Dystrophin-deficient mdx Mice by a Chimeric Peptide-PMO

HaiFang Yin et al.

MOLECULAR THERAPY (2010)

Article Biotechnology & Applied Microbiology

Targeted Delivery of siRNA to Macrophages for Anti-inflammatory Treatment

Sang-Soo Kim et al.

MOLECULAR THERAPY (2010)

Article Biotechnology & Applied Microbiology

Rational Design of Immunostimulatory siRNAs

Michael P. Gantier et al.

MOLECULAR THERAPY (2010)

News Item Biotechnology & Applied Microbiology

Safety signal dampens reception for mipomersen antisense

Jim Kling

NATURE BIOTECHNOLOGY (2010)

Article Biotechnology & Applied Microbiology

Rational design of cationic lipids for siRNA delivery

Sean C. Semple et al.

NATURE BIOTECHNOLOGY (2010)

Review Biotechnology & Applied Microbiology

Targeting microRNAs in cancer: rationale, strategies and challenges

Ramiro Garzon et al.

NATURE REVIEWS DRUG DISCOVERY (2010)

Article Biochemistry & Molecular Biology

A rapid and sensitive method to detect siRNA-mediated mRNA cleavage in vivo using 5' RACE and a molecular beacon probe

Annette Lasham et al.

NUCLEIC ACIDS RESEARCH (2010)

Review Biochemistry & Molecular Biology

Sequence-non-specific effects of RNA interference triggers and microRNA regulators

Marta Olejniczak et al.

NUCLEIC ACIDS RESEARCH (2010)

Article Biochemistry & Molecular Biology

Anti-tumor activity of splice-switching oligonucleotides

John A. Bauman et al.

NUCLEIC ACIDS RESEARCH (2010)

Article Biochemistry & Molecular Biology

Synergistic effects between analogs of DNA and RNA improve the potency of siRNA-mediated gene silencing

Glen F. Deleavey et al.

NUCLEIC ACIDS RESEARCH (2010)

Biographical-Item Biochemistry & Molecular Biology

Remembering Paul C. Zamecnik, MD, Father of Antisense (1912-2009) OBITUARY

[Anonymous]

OLIGONUCLEOTIDES (2010)

Article Multidisciplinary Sciences

Therapeutic Silencing of MicroRNA-122 in Primates with Chronic Hepatitis C Virus Infection

Robert E. Lanford et al.

SCIENCE (2010)

Review Pharmacology & Pharmacy

Lipid-based systemic delivery of siRNA

Yu-Cheng Tseng et al.

ADVANCED DRUG DELIVERY REVIEWS (2009)

Article Oncology

Modification of HER2 pre-mRNA alternative splicing and its effects on breast cancer cells

Jing Wan et al.

INTERNATIONAL JOURNAL OF CANCER (2009)

Article Medicine, Research & Experimental

Confirming the RNAi-mediated mechanism of action of siRNA-based cancer therapeutics in mice

Adam D. Judge et al.

JOURNAL OF CLINICAL INVESTIGATION (2009)

Editorial Material Clinical Neurology

Less is more: therapeutic exon skipping for Duchenne muscular dystrophy

Annemieke Aartsma-Rus et al.

LANCET NEUROLOGY (2009)

Article Biotechnology & Applied Microbiology

Asymmetric Shorter-duplex siRNA Structures Trigger Efficient Gene Silencing With Reduced Nonspecific Effects

Chan Il Chang et al.

MOLECULAR THERAPY (2009)

Review Multidisciplinary Sciences

On the road to reading the RNA-interference code

Haruhiko Siomi et al.

NATURE (2009)

Article Biotechnology & Applied Microbiology

In vivo delivery of siRNA to immune cells by conjugation to a TLR9 agonist enhances antitumor immune responses

Marcin Kortylewski et al.

NATURE BIOTECHNOLOGY (2009)

Article Biotechnology & Applied Microbiology

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

Justin P. Dassie et al.

NATURE BIOTECHNOLOGY (2009)

Article Biotechnology & Applied Microbiology

Efficient siRNA delivery into primary cells by a peptide transduction domain-dsRNA binding domain fusion protein

Akiko Eguchi et al.

NATURE BIOTECHNOLOGY (2009)

Correction Biotechnology & Applied Microbiology

Transfection of small RNAs globally perturbs gene regulation by endogenous microRNAs (vol 6, pg 549, 2009)

Aly A. Khan et al.

NATURE BIOTECHNOLOGY (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

Potent inhibition of microRNA in vivo without degradation

Scott Davis et al.

NUCLEIC ACIDS RESEARCH (2009)

Article Biochemistry & Molecular Biology

miR2Disease: a manually curated database for microRNA deregulation in human disease

Qinghua Jiang et al.

NUCLEIC ACIDS RESEARCH (2009)

Review Biochemistry & Molecular Biology

Therapeutic Potential of Splice-Switching Oligonucleotides

John Bauman et al.

OLIGONUCLEOTIDES (2009)

Review Biochemistry & Molecular Biology

siRNA and Innate Immunity

Marjorie Robbins et al.

OLIGONUCLEOTIDES (2009)

Review Biochemistry & Molecular Biology

Therapeutic Applications of DNA and RNA Aptamers

Kristina W. Thiel et al.

OLIGONUCLEOTIDES (2009)

Review Developmental Biology

A Primer for Morpholino Use in Zebrafish

Brent R. Bill et al.

ZEBRAFISH (2009)

Review Chemistry, Multidisciplinary

2′F-arabinonucleic acids (2′F-ANA) -: History, properties, and new frontiers

Jonathan K. Watts et al.

CANADIAN JOURNAL OF CHEMISTRY (2008)

Review Pharmacology & Pharmacy

Chemically modified siRNA: tools and applications

Jonathan K. Watts et al.

DRUG DISCOVERY TODAY (2008)

Review Biotechnology & Applied Microbiology

The business of RNAi therapeutics

Dirk Haussecker

HUMAN GENE THERAPY (2008)

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

Overcoming the innate immune response to small interfering RNA

Adam Judge et al.

HUMAN GENE THERAPY (2008)

Review Gastroenterology & Hepatology

miR-122, a paradigm for the role of microRNAs in the liver

Muriel Girard et al.

JOURNAL OF HEPATOLOGY (2008)

Article Biotechnology & Applied Microbiology

Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice

Natee Jearawiriyapaisarn et al.

MOLECULAR THERAPY (2008)

Article Multidisciplinary Sciences

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

Mark E. Kleinman et al.

NATURE (2008)

Article Multidisciplinary Sciences

LNA-mediated microRNA silencing in non-human primates

Joacim Elmen et al.

NATURE (2008)

Article Biotechnology & Applied Microbiology

Asymmetric RNA duplexes mediate RNA interference in mammalian cells

Xiangao Sun et al.

NATURE BIOTECHNOLOGY (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)

Article Biochemistry & Molecular Biology

5′-triphosphate-siRNA: turning gene silencing and Rig-I activation against melanoma

Hendrik Poeck et al.

NATURE MEDICINE (2008)

Article Biochemical Research Methods

Inhibition of microRNA with antisense oligonucleotides

Christine C. Esau

METHODS (2008)

Review Biochemistry & Molecular Biology

Mechanisms and strategies for effective delivery of antisense and siRNA oligonucleotides

Rudy Juliano et al.

NUCLEIC ACIDS RESEARCH (2008)

Article Biochemistry & Molecular Biology

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)

Article Biochemistry & Molecular Biology

Chemical modification patterns compatible with high potency Dicer-substrate small interfering RNAs

Michael A. Collingwood et al.

OLIGONUCLEOTIDES (2008)

Article Multidisciplinary Sciences

An Analysis of Human MicroRNA and Disease Associations

Ming Lu et al.

PLOS ONE (2008)

Article Multidisciplinary Sciences

Effects of local mRNA structure on posttranscriptional gene silencing

Stephen I. Rudnick et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2008)

Article Multidisciplinary Sciences

Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer

Bo Wu et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2008)

Review Oncology

Telomerase and cancer therapeutics

Calvin B. Harley

NATURE REVIEWS CANCER (2008)

Review Biotechnology & Applied Microbiology

MicroRNAs in disease and potential therapeutic applications

Harris S. Soifer et al.

MOLECULAR THERAPY (2007)

Review Medicine, Research & Experimental

Therapeutic application of RNAi: is mRNA targeting finally ready for prime time?

Dirk Grimm et al.

JOURNAL OF CLINICAL INVESTIGATION (2007)

Review Medicine, Research & Experimental

Nonviral delivery of synthetic siRNAs in vivo

Saghir Akhtar et al.

JOURNAL OF CLINICAL INVESTIGATION (2007)

Review Medicine, Research & Experimental

Chemical modification: the key to clinical application of RNA interference?

David R. Corey

JOURNAL OF CLINICAL INVESTIGATION (2007)

Article Biochemistry & Molecular Biology

Synthetic agonists of Toll-like receptors 7, 8 and 9

S. Agrawal et al.

BIOCHEMICAL SOCIETY TRANSACTIONS (2007)

Review Biochemistry & Molecular Biology

Antisense-mediated exon skipping: A versatile tool with therapeutic and research applications

Annemieke Aartsma-Rus et al.

Article Biochemistry & Molecular Biology

Improved silencing properties using small internally segmented interfering RNAs

Jesper B. Bramsen et al.

NUCLEIC ACIDS RESEARCH (2007)

Article Chemistry, Multidisciplinary

Five- and six-membered conformationally locked 2′,4′′-carbocyclic ribo-thymidines:: Synthesis, structure, and biochemical studies

Puneet Srivastava et al.

JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2007)

Article Biotechnology & Applied Microbiology

Antisense - down, but not out

Carol Potera

NATURE BIOTECHNOLOGY (2007)

Review Chemistry, Multidisciplinary

Lipid-based nanoparticles for nucleic acid delivery

Weijun Li et al.

PHARMACEUTICAL RESEARCH (2007)

Review Biochemistry & Molecular Biology

Hydrophobization and bioconjugation for enhanced siRNA delivery and targeting

Daniel De Paula et al.

Review Biotechnology & Applied Microbiology

siRNA and isRNA: Two edges of one sword

Martin Schlee et al.

MOLECULAR THERAPY (2006)

Article Biochemistry & Molecular Biology

Competition for RISC binding predicts in vitro potency of siRNA

Erich Koller et al.

NUCLEIC ACIDS RESEARCH (2006)

Review Microbiology

Aptamers come of age - at last

David H. J. Bunka et al.

NATURE REVIEWS MICROBIOLOGY (2006)

Article Biochemistry & Molecular Biology

Position-specific chemical modification of siRNAs reduces off-target'' transcript silencing

Aimee L. Jackson et al.

Article Biochemistry & Molecular Biology

Off-target effects by siRNA can induce toxic phenotype

Yuriy Fedorov et al.

Article Genetics & Heredity

LNA-modified oligonucleotides mediate specific inhibition of microRNA function

Ulf Andersson Orom et al.

Article Biotechnology & Applied Microbiology

Design of noninflammatory synthetic siRNA mediating potent gene silencing in vivo

AD Judge et al.

MOLECULAR THERAPY (2006)

Article Biochemistry & Molecular Biology

Synthesis and properties of 2′-O,4-C-methyleneoxymethylene bridged nucleic acid

Y Hari et al.

BIOORGANIC & MEDICINAL CHEMISTRY (2006)

Article Biochemistry & Molecular Biology

Plasma protein binding of an antisense oligonucleotide targeting human ICAM-1 (ISIS 2302)

Tanya A. Watanabe et al.

OLIGONUCLEOTIDES (2006)

Article Multidisciplinary Sciences

Silencing of microRNAs in vivo with 'antagomirs'

J Krützfeldt et al.

NATURE (2005)

Review Biotechnology & Applied Microbiology

Activation of the mammalian immune system by siRNAs

JT Marques et al.

NATURE BIOTECHNOLOGY (2005)

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

Design of a genome-wide siRNA library using an artificial neural network

D Huesken 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

Synthetic dsRNA Dicer substrates enhance RNAi potency and efficacy

DH Kim et al.

NATURE BIOTECHNOLOGY (2005)

Article Biochemistry & Molecular Biology

Determination of the role of the human RNase H1 in the pharmacology of DNA-like antisense drugs

HJ Wu et al.

JOURNAL OF BIOLOGICAL CHEMISTRY (2004)

Article Chemistry, Medicinal

Biodistribution of phosphodiester and phosphorothioate siRNA

DA Braasch et al.

BIOORGANIC & MEDICINAL CHEMISTRY LETTERS (2004)

Article Biochemistry & Molecular Biology

Sequence-specific inhibition of microRNA- and siRNA-induced RNA silencing

G Meister et al.

Review Biochemistry & Molecular Biology

MicroRNAs: Genomics, biogenesis, mechanism, and function

DP Bartel

Article Biochemistry & Molecular Biology

siRNA Selection Server: an automated siRNA oligonucleotide prediction server

BB Yuan et al.

NUCLEIC ACIDS RESEARCH (2004)

Review Chemistry, Medicinal

Development of bridged nucleic acid analogues for antigene technology

S Obika

CHEMICAL & PHARMACEUTICAL BULLETIN (2004)

Article Biotechnology & Applied Microbiology

Expression profiling reveals off-target gene regulation by RNAi

AL Jackson et al.

NATURE BIOTECHNOLOGY (2003)

Article Biotechnology & Applied Microbiology

Repair of a splicing defect in erythroid cells from patients with β-thalassemia/HbE disorder

T Suwanmanee et al.

MOLECULAR THERAPY (2002)

Article Biochemistry & Molecular Biology

Developments in RNA chemistry, a personal view

F Eckstein

BIOCHIMIE (2002)

Article Biochemistry & Molecular Biology

Antisense properties of tricyclo-DNA

D Renneberg et al.

NUCLEIC ACIDS RESEARCH (2002)

Review Biochemistry & Molecular Biology

DNA analogues: From supramolecular principles to biological properties

CJ Leumann

BIOORGANIC & MEDICINAL CHEMISTRY (2002)

Article Multidisciplinary Sciences

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

SM Elbashir et al.

NATURE (2001)

Review Pharmacology & Pharmacy

Fomivirsen

LR Grillone et al.

DRUGS OF TODAY (2001)

Article Cell Biology

RNA interference is mediated by 21-and 22-nucleotide RNAs

SM Elbashir et al.

GENES & DEVELOPMENT (2001)

Article Biochemistry & Molecular Biology

Locked nucleic acid (LNA): fine-tuning the recognition of DNA and RNA

DA Braasch et al.

CHEMISTRY & BIOLOGY (2001)

Article Multidisciplinary Sciences

Potent and nontoxic antisense oligonucleotides containing locked nucleic acids

C Wahlestedt et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2000)