Related references
Note: Only part of the references are listed.Functional Classification and Experimental Dissection of Long Noncoding RNAs
Florian Kopp et al.
CELL (2018)
A modular platform for targeted RNAi therapeutics
Ranit Kedmi et al.
NATURE NANOTECHNOLOGY (2018)
mRNA vaccines - a new era in vaccinology
Norbert Pardi et al.
NATURE REVIEWS DRUG DISCOVERY (2018)
Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia
S. L. Maude et al.
NEW ENGLAND JOURNAL OF MEDICINE (2018)
Nucleic Acid Therapies for Cystic Fibrosis
Shruti Sasaki et al.
NUCLEIC ACID THERAPEUTICS (2018)
An Investigational RNAi Therapeutic Targeting Glycolate Oxidase Reduces Oxalate Production in Models of Primary Hyperoxaluria
Abigail Liebow et al.
JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY (2017)
Cornerstones of CRISPR-Cas in drug discovery and therapy
Christof Fellmann et al.
NATURE REVIEWS DRUG DISCOVERY (2017)
Aptamers as targeted therapeutics: current potential and challenges
Jiehua Zhou et al.
NATURE REVIEWS DRUG DISCOVERY (2017)
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
Stephen Russell et al.
LANCET (2017)
Clinical Proof of Concept for a Novel Hepatocyte-Targeting GaINAc-siRNA Conjugate
Tracy S. Zimmermann et al.
MOLECULAR THERAPY (2017)
Zika virus protection by a single low-dose nucleoside-modified mRNA vaccination
Norbert Pardi et al.
NATURE (2017)
Worth the RISC?
Ken Garber
NATURE BIOTECHNOLOGY (2017)
The chemical evolution of oligonucleotide therapies of clinical utility
Anastasia Khvorova et al.
NATURE BIOTECHNOLOGY (2017)
Cellular uptake and trafficking of antisense oligonucleotides
Stanley T. Crooke et al.
NATURE BIOTECHNOLOGY (2017)
Overcoming cellular barriers for RNA therapeutics
Steven F. Dowdy
NATURE BIOTECHNOLOGY (2017)
Refining strategies to translate genome editing to the clinic
Tatjana I. Cornu et al.
NATURE MEDICINE (2017)
RNA-based recognition and targeting: sowing the seeds of specificity
Stanislaw A. Gorski et al.
NATURE REVIEWS MOLECULAR CELL BIOLOGY (2017)
Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy
K. J. Pasi et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
A Highly Durable RNAi Therapeutic Inhibitor of PCSK9
Kevin Fitzgerald et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
Axicabtagene Ciloleucel CAR T-Cell Therapy in Refractory Large B-Cell Lymphoma
S. S. Neelapu et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
R. S. Finkel et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
Asialoglycoprotein receptor 1 mediates productive uptake of N-acetylgalactosamine-conjugated and unconjugated phosphorothioate antisense oligonucleotides into liver hepatocytes
Michael Tanowitz et al.
NUCLEIC ACIDS RESEARCH (2017)
The Dimensions, Dynamics, and Relevance of the Mammalian Noncoding Transcriptome
Ira W. Deveson et al.
TRENDS IN GENETICS (2017)
Molecular Mechanisms of Antisense Oligonucleotides
Stanley T. Crooke
NUCLEIC ACID THERAPEUTICS (2017)
Effect of PCSK9 Inhibitors on Clinical Outcomes in Patients With Hypercholesterolemia: A Meta-Analysis of 35 Randomized Controlled Trials
Aris Karatasakis et al.
JOURNAL OF THE AMERICAN HEART ASSOCIATION (2017)
Preclinical and Clinical Advances of GalNAc-Decorated Nucleic Acid Therapeutics
Yuanyu Huang
MOLECULAR THERAPY-NUCLEIC ACIDS (2017)
CRISPR/Cas9-Based Genome Editing for Disease Modeling and Therapy: Challenges and Opportunities for Nonviral Delivery
Hong-Xia Wang et al.
CHEMICAL REVIEWS (2017)
Modified mRNA Vaccines Protect against Zika Virus Infection
Justin M. Richner et al.
CELL (2017)
CRISPR-Based Technologies for the Manipulation of Eukaryotic Genomes
Alexis C. Komor et al.
CELL (2017)
Harnessing RNAi-based nanomedicines for therapeutic gene silencing in B-cell malignancies
Shiri Weinstein et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2016)
In vivo gene editing in dystrophic mouse muscle and muscle stem cells
Mohammadsharif Tabebordbar et al.
SCIENCE (2016)
In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy
Christopher E. Nelson et al.
SCIENCE (2016)
Seq and You Will Find
Nicole Schonrock et al.
CURRENT GENE THERAPY (2016)
Embracing proteins: structural themes in aptamer-protein complexes
Amy D. Gelinas et al.
CURRENT OPINION IN STRUCTURAL BIOLOGY (2016)
Advances in RNAi therapeutic delivery to leukocytes using lipid nanoparticles
Srinivas Ramishetti et al.
JOURNAL OF DRUG TARGETING (2016)
Inhibition of Glycolate Oxidase With Dicer-substrate siRNA Reduces Calcium Oxalate Deposition in a Mouse Model of Primary Hyperoxaluria Type 1
Chaitali Dutta et al.
MOLECULAR THERAPY (2016)
Systemic RNA delivery to dendritic cells exploits antiviral defence for cancer immunotherapy
Lena M. Kranz et al.
NATURE (2016)
Translation efficiency of mRNAs is increased by antisense oligonucleotides targeting upstream open reading frames
Xue-hai Liang et al.
NATURE BIOTECHNOLOGY (2016)
Unique features of long non-coding RNA biogenesis and function
Jeffrey J. Quinn et al.
NATURE REVIEWS GENETICS (2016)
Docosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain
Mehran Nikan et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2016)
Nucleic Acid Modifications in Regulation of Gene Expression
Kai Chen et al.
CELL CHEMICAL BIOLOGY (2016)
siRNA Conjugates Carrying Sequentially Assembled Trivalent N-Acetylgalactosamine Linked Through Nucleosides Elicit Robust Gene Silencing In Vivo in Hepatocytes
Shigeo Matsuda et al.
ACS CHEMICAL BIOLOGY (2015)
PIWI-Interacting RNA: Its Biogenesis and Functions
Yuka W. Iwasaki et al.
ANNUAL REVIEW OF BIOCHEMISTRY, VOL 84 (2015)
Hepatocyte-Specific Delivery of siRNAs Conjugated to Novel Non-nucleosidic Trivalent N-Acetylgalactosamine Elicits Robust Gene Silencing in Vivo
Kallanthottathil G. Rajeev et al.
CHEMBIOCHEM (2015)
Manipulating the in vivo immune response by targeted gene knockdown
Judy Lieberman
CURRENT OPINION IN IMMUNOLOGY (2015)
The roles of CRISPR-Cas systems in adaptive immunity and beyond
Rodolphe Barrangou
CURRENT OPINION IN IMMUNOLOGY (2015)
Gene Knockdown by EpCAM Aptamer-siRNA Chimeras Suppresses Epithelial Breast Cancers and Their Tumor-Initiating Cells
Adi Gilboa-Geffen et al.
MOLECULAR CANCER THERAPEUTICS (2015)
mRNA: Fulfilling the Promise of Gene Therapy
Drew Weissman et al.
MOLECULAR THERAPY (2015)
Visualizing lipid-formulated siRNA release from endosomes and target gene knockdown
Anders Wittrup et al.
NATURE BIOTECHNOLOGY (2015)
Knocking down disease: a progress report on siRNA therapeutics
Anders Wittrup et al.
NATURE REVIEWS GENETICS (2015)
RNA-mediated epigenetic regulation of gene expression
Daniel Holoch et al.
NATURE REVIEWS GENETICS (2015)
The Noncoding RNA Revolution-Trashing Old Rules to Forge New Ones
Thomas R. Cech et al.
CELL (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)
Posttranscriptional Regulation of Gene Expression by Piwi Proteins and piRNAs
Toshiaki Watanabe et al.
MOLECULAR CELL (2014)
Multifunctional Aptamer-miRNA Conjugates for Targeted Cancer Therapy
Carla L. Esposito et al.
MOLECULAR THERAPY (2014)
Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype
Hao Yin et al.
NATURE BIOTECHNOLOGY (2014)
Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice
Thazha P. Prakash et al.
NUCLEIC ACIDS RESEARCH (2014)
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)
Beyond antibodies: New affinity reagents to unlock the proteome
Bridget Lollo et al.
PROTEOMICS (2014)
A Decade of Riboswitches
Alexander Serganov et al.
CELL (2013)
Image-based analysis of lipid nanoparticle-mediated siRNA delivery, intracellular trafficking and endosomal escape
Jerome Gilleron et al.
NATURE BIOTECHNOLOGY (2013)
Treatment of HCV Infection by Targeting MicroRNA
Harry L. A. Janssen et al.
NEW ENGLAND JOURNAL OF MEDICINE (2013)
Current progress on aptamer-targeted oligonucleotide therapeutics
Justin P. Dassie et al.
THERAPEUTIC DELIVERY (2013)
Roles for MicroRNAs in Conferring Robustness to Biological Processes
Margaret S. Ebert et al.
CELL (2012)
RNA therapeutics: beyond RNA interference and antisense oligonucleotides
Ryszard Kole et al.
NATURE REVIEWS DRUG DISCOVERY (2012)
Advances in human proteomics at high scale with the SOMAscan proteomics platform
Larry Gold et al.
NEW BIOTECHNOLOGY (2012)
Riboswitches and the RNA World
Ronald R. Breaker
COLD SPRING HARBOR PERSPECTIVES IN BIOLOGY (2012)
Structure-based druggability assessment - identifying suitable targets for small molecule therapeutics
Eric B. Fauman et al.
CURRENT OPINION IN CHEMICAL BIOLOGY (2011)
Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study
Sebahattin Cirak et al.
LANCET (2011)
CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III
Elitza Deltcheva et al.
NATURE (2011)
Highly Efficient Reprogramming to Pluripotency and Directed Differentiation of Human Cells with Synthetic Modified mRNA
Luigi Warren et al.
CELL STEM CELL (2010)
Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial
Frederick J. Raal et al.
LANCET (2010)
Aptamers as therapeutics
Anthony D. Keefe et al.
NATURE REVIEWS DRUG DISCOVERY (2010)
Desperately seeking microRNA targets
Marshall Thomas et al.
NATURE STRUCTURAL & MOLECULAR BIOLOGY (2010)
A Genome-wide RNAi Screen Identifies Multiple Synthetic Lethal Interactions with the Ras Oncogene
Ji Luo et al.
CELL (2009)
MicroRNAs: Target Recognition and Regulatory Functions
David P. Bartel
CELL (2009)
Identifying druggable disease-modifying gene products
Scott J. Dixon et al.
CURRENT OPINION IN CHEMICAL BIOLOGY (2009)
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)
Systemic administration of optimized aptamer-siRNA chimeras promotes regression of PSMA-expressing tumors
Justin P. Dassie et al.
NATURE BIOTECHNOLOGY (2009)
Incorporation of Pseudouridine Into mRNA Yields Superior Nonimmunogenic Vector With Increased Translational Capacity and Biological Stability
Katalin Kariko et al.
MOLECULAR THERAPY (2008)
Systemic leukocyte-directed siRNA delivery revealing cyclin D1 as an anti-inflammatory target
Dan Peer et al.
SCIENCE (2008)
Cancer proliferation gene discovery through functional Genomics
Michael R. Schlabach et al.
SCIENCE (2008)
Profiling essential genes in human mammary cells by multiplex RNAi screening
Jose M. Silva et al.
SCIENCE (2008)
CRISPR Interference Limits Horizontal Gene Transfer in Staphylococci by Targeting DNA
Luciano A. Marraffini et al.
SCIENCE (2008)
Identification of host proteins required for HIV infection through a functional genomic screen
Abraham L. Brass et al.
SCIENCE (2008)
Antisense inhibition of proprotein convertase subtilisin/kexin type 9 reduces serum LDL in hyperlipidemic mice
Mark J. Graham et al.
JOURNAL OF LIPID RESEARCH (2007)
CRISPR provides acquired resistance against viruses in prokaryotes
Rodolphe Barrangou et al.
SCIENCE (2007)
Selective gene silencing in activated leukocytes by targeting siRNAs to the integrin lymphocyte function-associated antigen-1
Dan Peer et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2007)
Activating gene expression in mammalian cells with promoter-targeted duplex RNAs
Bethany A. Janowski et al.
NATURE CHEMICAL BIOLOGY (2007)
Small dsRNAs induce transcriptional activation in human cells
Long-Cheng Li et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2006)
Cell type-specific delivery of siRNAs with aptamer-siRNA chimeras
James O. McNamara et al.
NATURE BIOTECHNOLOGY (2006)
Position-specific chemical modification of siRNAs reduces off-target'' transcript silencing
Aimee L. Jackson et al.
RNA (2006)
Silencing of microRNAs in vivo with 'antagomirs'
J Krützfeldt et al.
NATURE (2005)
Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs
DV Morrissey et al.
NATURE BIOTECHNOLOGY (2005)
MicroRNA expression profiles classify human cancers
J Lu et al.
NATURE (2005)
Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors
EW Song et al.
NATURE BIOTECHNOLOGY (2005)
RNA structure: the long and the short of it
SR Holbrook
CURRENT OPINION IN STRUCTURAL BIOLOGY (2005)
Sequence-dependent stimulation of the mammalian innate immune response by synthetic siRNA
AD Judge et al.
NATURE BIOTECHNOLOGY (2005)
A resource for large-scale RNA-interference-based screens in mammals
PJ Paddison et al.
NATURE (2004)
Genome-wide RNAi analysis of growth and viability in Drosophila cells
M Boutros et al.
SCIENCE (2004)
RNAi-mediated targeting of heterochromatin by the RITS complex
A Verdel et al.
SCIENCE (2004)
Identification of Hedgehog pathway components by RNAi in Drosophila cultured cells
L Lum et al.
SCIENCE (2003)
RNA interference targeting Fas protects mice from fulminant hepatitis
EW Song et al.
NATURE MEDICINE (2003)
Suppression of chemokine receptor expression by RNA interference allows for inhibition of HIV-1 replication
MA Martínez et al.
AIDS (2002)
RNA interference directed against viral and cellular targets inhibits human immunodeficiency virus type 1 replication
RM Surabhi et al.
JOURNAL OF VIROLOGY (2002)
Prevention of HIV-1 infection in human peripheral blood mononuclear cells by specific RNA interference
WS Park et al.
NUCLEIC ACIDS RESEARCH (2002)
Regulation of heterochromatic silencing and histone H3 lysine-9 methylation by RNAi
TA Volpe et al.
SCIENCE (2002)
Modulation of HIV-1 replication by RNA interference
JM Jacque et al.
NATURE (2002)
siRNA-directed inhibition of HIV-1 infection
CD Novina et al.
NATURE MEDICINE (2002)
Design of a targeted peptide nucleic acid prodrug to inhibit hepatic human microsomal triglyceride transfer protein expression in hepatocytes
EAL Biessen et al.
BIOCONJUGATE CHEMISTRY (2002)
Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells
SM Elbashir et al.
NATURE (2001)
Functional genomic analysis of C-elegans chromosome I by systematic RNA interference
AG Fraser et al.
NATURE (2000)
HIV gag mRNA transfection of dendritic cells (DC) delivers encoded antigen to MHC class I and II molecules, causes DC maturation, and induces a potent human in vitro primary immune response
D Weissman et al.
JOURNAL OF IMMUNOLOGY (2000)
dsRNA-mediated gene silencing in cultured Drosophila cells:: a tissue culture model for the analysis of RNA interference
NJ Caplen et al.
GENE (2000)
Share Paper
