Artificial miRNAs targeting CAG repeat expansion in ORFs cause rapid deadenylation and translation inhibition of mutant transcripts

Generation of New Isogenic Models of Huntington’s Disease Using CRISPR-Cas9 Technology

(2020) Magdalena Dabrowska et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

The current state and future directions of RNAi-based therapeutics

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

Length of Uninterrupted CAG, Independent of Polyglutamine Size, Results in Increased Somatic Instability, Hastening Onset of Huntington Disease

(2019) Galen E.B. Wright et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Targeting Huntingtin Expression in Patients with Huntington’s Disease

(2019) Sarah J. Tabrizi et al.   NEW ENGLAND JOURNAL OF MEDICINE

Phenotype onset in Huntington’s disease knock‐in mice is correlated with the incomplete splicing of the mutant huntingtin gene

(2019) Nicholas R. Franich et al.   JOURNAL OF NEUROSCIENCE RESEARCH

Allele-selective transcriptional repression of mutant HTT for the treatment of Huntington’s disease

(2019) Bryan Zeitler et al.   NATURE MEDICINE

A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system

(2019) Julia F. Alterman et al.   NATURE BIOTECHNOLOGY

Suppression of Mutant Protein Expression in SCA3 and SCA1 Mice Using a CAG Repeat-Targeting Antisense Oligonucleotide

(2019) Eleni Kourkouta et al.   Molecular Therapy-Nucleic Acids

Limits of using oligonucleotides for allele-selective inhibition at trinucleotide repeat sequences – targeting the CAG repeat within ataxin-1

(2019) Jiaxin Hu et al.   NUCLEOSIDES NUCLEOTIDES & NUCLEIC ACIDS

Universal RNAi Triggers for the Specific Inhibition of Mutant Huntingtin, Atrophin-1, Ataxin-3, and Ataxin-7 Expression

(2019) Anna Kotowska-Zimmer et al.   Molecular Therapy-Nucleic Acids

Metazoan MicroRNAs

(2018) David P. Bartel   CELL

Mechanistic Insights into MicroRNA-Mediated Gene Silencing

(2018) Thomas F. Duchaine et al.   Cold Spring Harbor Perspectives in Biology

Precise Excision of the CAG Tract from the Huntingtin Gene by Cas9 Nickases

(2018) Magdalena Dabrowska et al.   Frontiers in Neuroscience

Regulation of microRNA function in animals

(2018) Luca F. R. Gebert et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

A novel class of microRNA-recognition elements that function only within open reading frames

(2018) Kai Zhang et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

Nuclear Localization of Huntingtin mRNA Is Specific to Cells of Neuronal Origin

(2018) Marie-Cécile Didiot et al.   Cell Reports

Revisiting the Closed-Loop Model and the Nature of mRNA 5′–3′ Communication

(2018) Quentin Vicens et al.   MOLECULAR CELL

Subcellular Heterogeneity of the microRNA Machinery

(2018) Michele Trabucchi   TRENDS IN GENETICS

CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington’s disease

(2017) Su Yang et al.   JOURNAL OF CLINICAL INVESTIGATION

Therapies targeting DNA and RNA in Huntington's disease

(2017) Edward J Wild et al.   LANCET NEUROLOGY

CRISPR/Cas9 Editing of the Mutant Huntingtin Allele In Vitro and In Vivo

(2017) Alex Mas Monteys et al.   MOLECULAR THERAPY

Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems

(2017) Ken Y Chan et al.   NATURE NEUROSCIENCE

Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs

(2017) Xiulong Shen et al.   NUCLEIC ACIDS RESEARCH

Reduction of Huntington’s Disease RNA Foci by CAG Repeat-Targeting Reagents

(2017) Martyna O. Urbanek et al.   Frontiers in Cellular Neuroscience

Structural Characteristics of Simple RNA Repeats Associated with Disease and their Deleterious Protein Interactions

(2017) Adam Ciesiolka et al.   Frontiers in Cellular Neuroscience

Widespread Translational Remodeling during Human Neuronal Differentiation

(2017) John D. Blair et al.   Cell Reports

The expanded CAG repeat in the huntingtin gene as target for therapeutic RNA modulation throughout the HD mouse brain

(2017) Nicole A. Datson et al.   PLoS One

The pathogenic exon 1 HTT protein is produced by incomplete splicing in Huntington’s disease patients

(2017) Andreas Neueder et al.   Scientific Reports

Silencing of genes responsible for polyQ diseases using chemically modified single-stranded siRNAs

(2016) Agnieszka Fiszer et al.   ACTA BIOCHIMICA POLONICA

The Biology of Huntingtin

(2016) Frédéric Saudou et al.   NEURON

Analysis of translation using polysome profiling

(2016) Héloïse Chassé et al.   NUCLEIC ACIDS RESEARCH

The DDX6–4E-T interaction mediates translational repression and P-body assembly

(2016) Anastasiia Kamenska et al.   NUCLEIC ACIDS RESEARCH

Stable association of RNAi machinery is conserved between the cytoplasm and nucleus of human cells

(2016) Roya Kalantari et al.   RNA

Mutant CAG Repeats Effectively Targeted by RNA Interference in SCA7 Cells

(2016) Agnieszka Fiszer et al.   Genes

Argonaute Family Protein Expression in Normal Tissue and Cancer Entities

(2016) Daniel Völler et al.   PLoS One

A Dynamic Search Process Underlies MicroRNA Targeting

(2015) Stanley D. Chandradoss et al.   CELL

In Vitro Expansion of CAG, CAA, and Mixed CAG/CAA Repeats

(2015) Grzegorz Figura et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Towards a molecular understanding of microRNA-mediated gene silencing

(2015) Stefanie Jonas et al.   NATURE REVIEWS GENETICS

Biochemical isolation of Argonaute protein complexes by Ago-APP

(2015) Judith Hauptmann et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Tissue-based map of the human proteome

(2015) M. Uhlen et al.   SCIENCE

Allele-Selective Inhibition of Mutant Atrophin-1 Expression by Duplex and Single-Stranded RNAs

(2014) Jiaxin Hu et al.   BIOCHEMISTRY

MicroRNA binding sites in the coding region of mRNAs: Extending the repertoire of post-transcriptional gene regulation

(2014) Anneke Brümmer et al.   BIOESSAYS

Assembly and function of small RNA – Argonaute protein complexes

(2014) Anne Dueck et al.   BIOLOGICAL CHEMISTRY

The panorama of miRNA-mediated mechanisms in mammalian cells

(2014) Anna Stroynowska-Czerwinska et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

MicroRNAs Block Assembly of eIF4F Translation Initiation Complex in Drosophila

(2014) Takashi Fukaya et al.   MOLECULAR CELL

MicroRNAs Trigger Dissociation of eIF4AI and eIF4AII from Target mRNAs in Humans

(2014) Akira Fukao et al.   MOLECULAR CELL

mRNA Destabilization Is the Dominant Effect of Mammalian MicroRNAs by the Time Substantial Repression Ensues

(2014) Stephen W. Eichhorn et al.   MOLECULAR CELL

Structural and Biochemical Insights to the Role of the CCR4-NOT Complex and DDX6 ATPase in MicroRNA Repression

(2014) Hansruedi Mathys et al.   MOLECULAR CELL

Oligonucleotide-based strategies to combat polyglutamine diseases

(2014) A. Fiszer et al.   NUCLEIC ACIDS RESEARCH

The role of RNA structure at 5′ untranslated region in microRNA-mediated gene regulation

(2014) Wanjun Gu et al.   RNA

Human DDX6 effects miRNA-mediated gene silencing via direct binding to CNOT1

(2014) Christopher Rouya et al.   RNA

Target Gene Abundance Contributes to the Efficiency of siRNA-Mediated Gene Silencing

(2014) Sun Woo Hong et al.   Nucleic Acid Therapeutics

Exploring the Effect of Sequence Length and Composition on Allele-Selective Inhibition of Human Huntingtin Expression by Single-Stranded Silencing RNAs

(2014) Jiaxin Hu et al.   Nucleic Acid Therapeutics

Assessment of Selective mRNA Translation in Mammalian Cells by Polysome Profiling

(2014) Mame Daro Faye et al.   Jove-Journal of Visualized Experiments

Allele-Selective Inhibition of Expression of Huntingtin and Ataxin-3 by RNA Duplexes Containing Unlocked Nucleic Acid Substitutions

(2013) Yuichiro Aiba et al.   BIOCHEMISTRY

Mapping the Human miRNA Interactome by CLASH Reveals Frequent Noncanonical Binding

(2013) Aleksandra Helwak et al.   CELL

ss-siRNAs allele selectively inhibit ataxin-3 expression: multiple mechanisms for an alternative gene silencing strategy

(2013) Jing Liu et al.   NUCLEIC ACIDS RESEARCH

RNA duplexes with abasic substitutions are potent and allele-selective inhibitors of huntingtin and ataxin-3 expression

(2013) J. Liu et al.   NUCLEIC ACIDS RESEARCH

Self-duplexing CUG repeats selectively inhibit mutant huntingtin expression

(2013) Agnieszka Fiszer et al.   NUCLEIC ACIDS RESEARCH

Translational Repression and eIF4A2 Activity Are Critical for MicroRNA-Mediated Gene Regulation

(2013) H. A. Meijer et al.   SCIENCE

Single-Stranded RNAs Use RNAi to Potently and Allele-Selectively Inhibit Mutant Huntingtin Expression

(2012) Dongbo Yu et al.   CELL

Polyglutamine neurodegeneration: expanded glutamines enhance native functions

(2012) Harry T Orr   CURRENT OPINION IN GENETICS & DEVELOPMENT

Allele-selective inhibition of trinucleotide repeat genes

(2012) Masayuki Matsui et al.   DRUG DISCOVERY TODAY

Kinetic analysis reveals successive steps leading to miRNA-mediated silencing in mammalian cells

(2012) Julien Béthune et al.   EMBO REPORTS

Quantitative functions of Argonaute proteins in mammalian development

(2012) D. Wang et al.   GENES & DEVELOPMENT

An anatomically comprehensive atlas of the adult human brain transcriptome

(2012) Michael J. Hawrylycz et al.   NATURE

The mechanics of miRNA-mediated gene silencing: a look under the hood of miRISC

(2012) Marc R Fabian et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

Mechanism of allele-selective inhibition of huntingtin expression by duplex RNAs that target CAG repeats: function through the RNAi pathway

(2012) Jiaxin Hu et al.   NUCLEIC ACIDS RESEARCH

The interactions of GW182 proteins with PABP and deadenylases are required for both translational repression and degradation of miRNA targets

(2012) Eric Huntzinger et al.   NUCLEIC ACIDS RESEARCH

microRNAs associated with the different human Argonaute proteins

(2012) A. Dueck et al.   NUCLEIC ACIDS RESEARCH

The role of the 3' untranslated region in post-transcriptional regulation of protein expression in mammalian cells.

(2012) Eva Matoulkova et al.   RNA Biology

miRNA-Mediated Gene Silencing by Translational Repression Followed by mRNA Deadenylation and Decay

(2012) S. Djuranovic et al.   SCIENCE

Allele-selective inhibition of ataxin-3 (ATX3) expression by antisense oligomers and duplex RNAs

(2011) Jiaxin Hu et al.   BIOLOGICAL CHEMISTRY

The code within the code: microRNAs target coding regions

(2011) Joshua J. Forman et al.   CELL CYCLE

Unusually effective microRNA targeting within repeat-rich coding regions of mammalian mRNAs

(2011) M. Schnall-Levin et al.   GENOME RESEARCH

GW182 Proteins Directly Recruit Cytoplasmic Deadenylase Complexes to miRNA Targets

(2011) Joerg E. Braun et al.   MOLECULAR CELL

miRNA-mediated deadenylation is orchestrated by GW182 through two conserved motifs that interact with CCR4–NOT

(2011) Marc R Fabian et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

miRNA repression involves GW182-mediated recruitment of CCR4–NOT through conserved W-containing motifs

(2011) Marina Chekulaeva et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

Inhibition of mutant huntingtin expression by RNA duplex targeting expanded CAG repeats

(2011) Agnieszka Fiszer et al.   NUCLEIC ACIDS RESEARCH

The Impact of miRNA Target Sites in Coding Sequences and in 3′UTRs

(2011) Zhuo Fang et al.   PLoS One

Transcriptome-wide Identification of RNA-Binding Protein and MicroRNA Target Sites by PAR-CLIP

(2010) Markus Hafner et al.   CELL

Allele-Selective Inhibition of Huntingtin Expression by Switching to an miRNA-like RNAi Mechanism

(2010) Jiaxin Hu et al.   CHEMISTRY & BIOLOGY

mRNA turnover rate limits siRNA and microRNA efficacy

(2010) Erik Larsson et al.   Molecular Systems Biology

Mammalian microRNAs predominantly act to decrease target mRNA levels

(2010) Huili Guo et al.   NATURE

MicroRNA-181a modulates gene expression of zinc finger family members by directly targeting their coding regions

(2010) Shenglin Huang et al.   NUCLEIC ACIDS RESEARCH

eIF5A Promotes Translation Elongation, Polysome Disassembly and Stress Granule Assembly

(2010) Chi Ho Li et al.   PLoS One

MicroRNA mir-346 targets the 5′-untranslated region of receptor-interacting protein 140 (RIP140) mRNA and up-regulates its protein expression

(2009) Nien-Pei Tsai et al.   BIOCHEMICAL JOURNAL

CRD-BP Protects the Coding Region of βTrCP1 mRNA from miR-183-Mediated Degradation

(2009) Irina Elcheva et al.   MOLECULAR CELL

Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs

(2009) Jiaxin Hu et al.   NATURE BIOTECHNOLOGY

Biological basis for restriction of microRNA targets to the 3′ untranslated region in mammalian mRNAs

(2009) Shuo Gu et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

MicroRNA-10a Binds the 5′UTR of Ribosomal Protein mRNAs and Enhances Their Translation

(2008) Ulf Andersson Ørom et al.   MOLECULAR CELL

MicroRNAs to Nanog, Oct4 and Sox2 coding regions modulate embryonic stem cell differentiation

(2008) Yvonne Tay et al.   NATURE

A search for conserved sequences in coding regions reveals that the let-7 microRNA targets Dicer within its coding sequence

(2008) J. J. Forman et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Deadenylation is a widespread effect of miRNA regulation

(2008) A. Eulalio et al.   RNA

miR-148 targets human DNMT3b protein coding region

(2008) A. M. Duursma et al.   RNA