4.3 Article

Oligonucleotides Targeting DNA Repeats Downregulate Huntingtin Gene Expression in Huntington's Patient-Derived Neural Model System

Related references

Note: Only part of the references are listed.
Article Clinical Neurology

Huntington's disease brain-derived small RNAs recapitulate associated neuropathology in mice

Jordi Creus-Muncunill et al.

Summary: While most research on Huntington's disease has focused on the pathogenic effects of proteins, growing evidence indicates that small RNAs produced in the putamen of HD patients can induce HD pathology in vivo. Different species of small RNAs, including sCAG and tRNA fragments, contribute to striatal dysfunction and general transcriptomic changes, highlighting the potential for therapeutic strategies based on blocking sRNA-mediated toxicity.

ACTA NEUROPATHOLOGICA (2021)

Article Biochemistry & Molecular Biology

Deregulated Splicing Is a Major Mechanism of RNA-Induced Toxicity in Huntington's Disease

Judith Schilling et al.

JOURNAL OF MOLECULAR BIOLOGY (2019)

Article Medicine, General & Internal

Targeting Huntingtin Expression in Patients with Huntington's Disease

Sarah J. Tabrizi et al.

NEW ENGLAND JOURNAL OF MEDICINE (2019)

Review Clinical Neurology

Targeted Oligonucleotides for Treating Neurodegenerative Tandem Repeat Diseases

Rula Zain et al.

NEUROTHERAPEUTICS (2019)

Article Biochemistry & Molecular Biology

Delivery of miRNA-Targeted Oligonucleotides in the Rat Striatum by Magnetofection with Neuromag®

Simoneide Souza Titze de Almeida et al.

MOLECULES (2018)

Review Biotechnology & Applied Microbiology

Oligonucleotide therapies for disorders of the nervous system

Olga Khorkova et al.

NATURE BIOTECHNOLOGY (2017)

Article Biochemistry & Molecular Biology

CTG repeat-targeting oligonucleotides for down-regulating Huntingtin expression

Eman M. Zaghloul et al.

NUCLEIC ACIDS RESEARCH (2017)

Review Genetics & Heredity

Ataxin-2: From RNA Control to Human Health and Disease

Lauren A. Ostrowski et al.

GENES (2017)

Article Medicine, Research & Experimental

Targeting CAG repeat RNAs reduces Huntington's disease phenotype independently of huntingtin levels

Laura Rue et al.

JOURNAL OF CLINICAL INVESTIGATION (2016)

Article Cell & Tissue Engineering

Cell Polarity and Neurogenesis in Embryonic Stem Cell-Derived Neural Rosettes

Erin Banda et al.

STEM CELLS AND DEVELOPMENT (2015)

Article Neurosciences

Oligonucleotide-based therapy for neurodegenerative diseases

Iddo Magen et al.

BRAIN RESEARCH (2014)

Review Developmental Biology

The roles and regulation of multicellular rosette structures during morphogenesis

Molly J. Harding et al.

DEVELOPMENT (2014)

Article Biochemistry & Molecular Biology

Development of bis-locked nucleic acid (bisLNA) oligonucleotides for efficient invasion of supercoiled duplex DNA

Pedro M. D. Moreno et al.

NUCLEIC ACIDS RESEARCH (2013)

Article Cell & Tissue Engineering

Genetic Correction of Huntington's Disease Phenotypes in Induced Pluripotent Stem Cells

Mahru C. An et al.

CELL STEM CELL (2012)

Article Biochemical Research Methods

Fiji: an open-source platform for biological-image analysis

Johannes Schindelin et al.

NATURE METHODS (2012)

Article Cell & Tissue Engineering

hESC derived neuro-epithelial rosettes recapitulate early mammalian neurulation events; an in vitro model

Carol Lynn Curchoe et al.

STEM CELL RESEARCH (2012)

Article Biotechnology & Applied Microbiology

Preclinical Safety of RNAi-Mediated HTT Suppression in the Rhesus Macaque as a Potential Therapy for Huntington's Disease

Jodi L. McBride et al.

MOLECULAR THERAPY (2011)

Article Biochemistry & Molecular Biology

Mutant CAG repeats of Huntingtin transcript fold into hairpins, form nuclear foci and are targets for RNA interference

Mateusz de Mezer et al.

NUCLEIC ACIDS RESEARCH (2011)

Article Biochemistry & Molecular Biology

A Model for Neural Development and Treatment of Rett Syndrome Using Human Induced Pluripotent Stem Cells

Maria C. N. Marchetto et al.

Article Multidisciplinary Sciences

Induced pluripotent stem cells from a spinal muscular atrophy patient

Allison D. Ebert et al.

NATURE (2009)

Article Multidisciplinary Sciences

Neurons derived from reprogrammed fibroblasts functionally integrate into the fetal brain and improve symptoms of rats with Parkinson's disease

Marius Wernig et al.

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

Article Multidisciplinary Sciences

Reprogramming of human somatic cells to pluripotency with defined factors

In-Hyun Park et al.

NATURE (2008)

Article Multidisciplinary Sciences

Induced pluripotent stem cell lines derived from human somatic cells

Junying Yu et al.

SCIENCE (2007)

Article Genetics & Heredity

Factors associated with HD CAG repeat instability in Huntington disease

V. C. Wheeler et al.

JOURNAL OF MEDICAL GENETICS (2007)

Article Biochemical Research Methods

Efficient transfection of DNA or shRNA vectors into neurons using magnetofection

Thomas Buerli et al.

NATURE PROTOCOLS (2007)

Article Biochemistry & Molecular Biology

Dramatic tissue-specific mutation length increases are an early molecular event in Huntington disease pathogenesis

L Kennedy et al.

HUMAN MOLECULAR GENETICS (2003)

Article Multidisciplinary Sciences

Transcriptional regulation of cortical neuron migration by POU domain factors

RJ McEvilly et al.

SCIENCE (2002)

Article Biochemistry & Molecular Biology

Magnetofection: enhancing and targeting gene delivery by magnetic force in vitro and in vivo

F Scherer et al.

GENE THERAPY (2002)

Article Biochemistry & Molecular Biology

Fully modified 2′ MOE oligonucleotides redirect polyadenylation

TA Vickers et al.

NUCLEIC ACIDS RESEARCH (2001)