4.8 Article

Potent and sustained huntingtin lowering via AAV5 encoding miRNA preserves striatal volume and cognitive function in a humanized mouse model of Huntington disease

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。
Article Medicine, Research & Experimental

AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models

Elisabeth A. Spronck et al.

MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (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)

Article Biochemistry & Molecular Biology

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

Bryan Zeitler et al.

NATURE MEDICINE (2019)

Article Biotechnology & Applied Microbiology

Artificial miRNAs Reduce Human Mutant Huntingtin Throughout the Striatum in a Transgenic Sheep Model of Huntington's Disease

Edith L. Pfister et al.

HUMAN GENE THERAPY (2018)

Review Biotechnology & Applied Microbiology

Translation of MicroRNA-Based Huntingtin-Lowering Therapies from Preclinical Studies to the Clinic

Jana Miniarikova et al.

MOLECULAR THERAPY (2018)

Review Biotechnology & Applied Microbiology

Therapeutic approaches to Huntington disease: from the bench to the clinic

Nicholas S. Caron et al.

NATURE REVIEWS DRUG DISCOVERY (2018)

Article Cell Biology

Huntingtin suppression restores cognitive function in a mouse model of Huntington's disease

Amber L. Southwell et al.

SCIENCE TRANSLATIONAL MEDICINE (2018)

Article Biochemistry & Molecular Biology

The targetable A1 Huntington disease haplotype has distinct Amerindian and European origins in Latin America

Chris Kay et al.

EUROPEAN JOURNAL OF HUMAN GENETICS (2017)

Article Biochemistry & Molecular Biology

MR-guided parenchymal delivery of adeno-associated viral vector serotype 5 in non-human primate brain

L. Samaranch et al.

GENE THERAPY (2017)

Article Neurosciences

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

Martyna O. Urbanek et al.

FRONTIERS IN CELLULAR NEUROSCIENCE (2017)

Article Biotechnology & Applied Microbiology

Persistent Expression of Dopamine-Synthesizing Enzymes 15 Years After Gene Transfer in a Primate Model of Parkinson's Disease

Yoshihide Sehara et al.

HUMAN GENE THERAPY CLINICAL DEVELOPMENT (2017)

Article Biochemistry & Molecular Biology

A huntingtin-mediated fast stress response halting endosomal trafficking is defective in Huntington's disease

Siddharth Nath et al.

HUMAN MOLECULAR GENETICS (2015)

Article Biotechnology & Applied Microbiology

In Vivo Evaluation of Candidate Allele-specific Mutant Huntingtin Gene Silencing Antisense Oligonucleotides

Amber L. Southwell et al.

MOLECULAR THERAPY (2014)

Article Clinical Neurology

Multisource Ascertainment of Huntington Disease in Canada: Prevalence and Population at Risk

Emily R. Fisher et al.

MOVEMENT DISORDERS (2014)

Article Multidisciplinary Sciences

Allele-Specific Silencing of Mutant Huntingtin in Rodent Brain and Human Stem Cells

Valerie Drouet et al.

PLOS ONE (2014)

Article Biochemistry & Molecular Biology

Huntington disease in the South African population occurs on diverse and ethnically distinct genetic haplotypes

Fiona K. Baine et al.

EUROPEAN JOURNAL OF HUMAN GENETICS (2013)

Article Biochemical Research Methods

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

Johannes Schindelin et al.

NATURE METHODS (2012)

Review Biochemistry & Molecular Biology

Antisense oligonucleotide therapeutics for inherited neurodegenerative diseases

Amber L. Southwell et al.

TRENDS IN MOLECULAR MEDICINE (2012)

Article Biochemistry & Molecular Biology

HTT haplotypes contribute to differences in Huntington disease prevalence between Europe and East Asia

Simon C. Warby et al.

EUROPEAN JOURNAL OF HUMAN GENETICS (2011)

Article Multidisciplinary Sciences

Targeting Several CAG Expansion Diseases by a Single Antisense Oligonucleotide

Melvin M. Evers et al.

PLOS ONE (2011)

Article Biochemistry & Molecular Biology

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

Jiaxin Hu et al.

CHEMISTRY & BIOLOGY (2010)

Article Genetics & Heredity

CAG Expansion in the Huntington Disease Gene Is Associated with a Specific and Targetable Predisposing Haplogroup

Simon C. Warby et al.

AMERICAN JOURNAL OF HUMAN GENETICS (2009)

Article Clinical Neurology

Sustained Effects of Nonallele-Specific Huntingtin Silencing

Valerie Drouet et al.

ANNALS OF NEUROLOGY (2009)

Article Biochemistry & Molecular Biology

Mutant Huntingtin Impairs Vesicle Formation from Recycling Endosomes by Interfering with Rab11 Activity

Xueyi Li et al.

MOLECULAR AND CELLULAR BIOLOGY (2009)

Article Biotechnology & Applied Microbiology

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

Jiaxin Hu et al.

NATURE BIOTECHNOLOGY (2009)

Article Multidisciplinary Sciences

Expression of mutant huntingtin in mouse brain astrocytes causes age-dependent neurological symptoms

Jennifer Bradford et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2009)

Article Biotechnology & Applied Microbiology

Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection

Carmela Zincarelli et al.

MOLECULAR THERAPY (2008)

Article Multidisciplinary Sciences

Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi

Jodi L. McBride et al.

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

Article Biochemistry & Molecular Biology

Huntingtin has a membrane association signal that can modulate huntingtin aggregation, nuclear entry and toxicity

Randy Singh Atwal et al.

HUMAN MOLECULAR GENETICS (2007)

Article Multidisciplinary Sciences

Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits

M. DiFiglia et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2007)

Article Biotechnology & Applied Microbiology

CAG-encoded polyglutamine length polymorphism in the human genome

Stefanie L. Butland et al.

BMC GENOMICS (2007)

Article Biochemistry & Molecular Biology

Mutant huntingtin inhibits clathrin-independent endocytosis and causes accumulation of cholesterol in vitro and in vivo

Eugenia Trushina et al.

HUMAN MOLECULAR GENETICS (2006)

Article Biochemistry & Molecular Biology

rAAV-mediated shRNA ameliorated neuropathology in Huntington disease model mouse

Y Machida et al.

BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS (2006)

Article Neurosciences

Clinico-pathological rescue of a model mouse of Huntington's disease by siRNA

YL Wang et al.

NEUROSCIENCE RESEARCH (2005)

Article Biochemistry & Molecular Biology

Mitochondrial respiration and ATP production are significantly impaired in striatal cells expressing mutant Huntingtin

T Milakovic et al.

JOURNAL OF BIOLOGICAL CHEMISTRY (2005)

Article Multidisciplinary Sciences

RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model

SQ Harper et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2005)

Article Biochemistry & Molecular Biology

Mutant huntingtin impairs axonal trafficking in mammalian neurons in vivo and in vitro

E Trushina et al.

MOLECULAR AND CELLULAR BIOLOGY (2004)

Article Multidisciplinary Sciences

Loss of huntingtin-mediated BDNF gene transcription in Huntington's disease

C Zuccato et al.

SCIENCE (2001)