(Q45304275)

13 June 2018

title

Therapies targeting DNA and RNA in Huntington's disease (English)

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13 June 2018

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2

Sarah J Tabrizi

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13 June 2018

author name string

Edward J Wild

series ordinal

1

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13 June 2018

publication date

12 September 2017

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13 June 2018

published in

Lancet Neurology

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13 June 2018

volume

16

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13 June 2018

issue

10

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13 June 2018

page(s)

837-847

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13 June 2018

Neurofilament light protein in blood as a potential biomarker of neurodegeneration in Huntington's disease: a retrospective cohort analysis

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

14 June 2018

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14 June 2018

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Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy

14 June 2018

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Cerebrospinal Fluid Biomarkers for Huntington's Disease

14 June 2018

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Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntingtin for Development of Gene Therapy for Huntington's Disease.

14 June 2018

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14 June 2018

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Genome engineering through CRISPR/Cas9 technology in the human germline and pluripotent stem cells

14 June 2018

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Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain

14 June 2018

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RAN Translation in Huntington Disease

14 June 2018

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Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia

14 June 2018

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Advances in therapeutic CRISPR/Cas9 genome editing

14 June 2018

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Huntingtin Haplotypes Provide Prioritized Target Panels for Allele-specific Silencing in Huntington Disease Patients of European Ancestry.

14 June 2018

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Quantification of mutant huntingtin protein in cerebrospinal fluid from Huntington's disease patients

14 June 2018

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Therapeutic genome editing: prospects and challenges

14 June 2018

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Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy

14 June 2018

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Neuronal targets for reducing mutant huntingtin expression to ameliorate disease in a mouse model of Huntington's disease

14 June 2018

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Huntington disease: natural history, biomarkers and prospects for therapeutics

14 June 2018

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Increased brain penetration and potency of a therapeutic antibody using a monovalent molecular shuttle

14 June 2018

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Choosing an animal model for the study of Huntington's disease

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Predictors of phenotypic progression and disease onset in premanifest and early-stage Huntington's disease in the TRACK-HD study: analysis of 36-month observational data

14 June 2018

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study

14 June 2018

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Aberrant splicing of HTT generates the pathogenic exon 1 protein in Huntington disease

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression.

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis.

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

"Huntingtin holiday": progress toward an antisense therapy for Huntington's disease

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Common SNP-based haplotype analysis of the 4p16.3 Huntington disease gene region

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

CAG repeat expansion in Huntington disease determines age at onset in a fully dominant fashion.

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Potential endpoints for clinical trials in premanifest and early Huntington's disease in the TRACK-HD study: analysis of 24 month observational data

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Targeting several CAG expansion diseases by a single antisense oligonucleotide

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Immune responses to AAV in clinical trials

14 June 2018

1 reference

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Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Huntington's disease: from molecular pathogenesis to clinical treatment

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Biological and clinical changes in premanifest and early stage Huntington's disease in the TRACK-HD study: the 12-month longitudinal analysis

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

The discovery of zinc fingers and their applications in gene regulation and genome manipulation.

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Therapeutic interventions for disease progression in Huntington's disease

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Linking SNPs to CAG repeat length in Huntington's disease patients.

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

AAV vector-mediated RNAi of mutant huntingtin expression is neuroprotective in a novel genetic rat model of Huntington's disease

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Argonaute proteins: key players in RNA silencing

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Phylogenetic comparison of huntingtin homologues reveals the appearance of a primitive polyQ in sea urchin.

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

CAG-encoded polyglutamine length polymorphism in the human genome

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Antisense oligonucleotides: from design to therapeutic application

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

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

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Role of Toll-like receptors in antisense and siRNA [corrected].

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Huntingtin controls neurotrophic support and survival of neurons by enhancing BDNF vesicular transport along microtubules

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Inactivation of Hdh in the brain and testis results in progressive neurodegeneration and sterility in mice

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Exon 1 of the HD gene with an expanded CAG repeat is sufficient to cause a progressive neurological phenotype in transgenic mice

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Targeted disruption of the Huntington's disease gene results in embryonic lethality and behavioral and morphological changes in heterozygotes

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

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

14 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Deimmunization for gene therapy: host matching of synthetic zinc finger constructs enables long-term mutant Huntingtin repression in mice

16 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Huntingtin's spherical solenoid structure enables polyglutamine tract-dependent modulation of its structure and function

16 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

The incidence and prevalence of Huntington's disease: a systematic review and meta-analysis

16 June 2018

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Six-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum

16 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

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

16 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5604739

Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR/Cas9.

15 August 2018

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Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study

15 August 2018

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15 August 2018

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15 August 2018

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7 January 2021

Widespread suppression of huntingtin with convection-enhanced delivery of siRNA

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7 January 2021

RNase H-mediated inhibition of translation by antisense oligodeoxyribonucleotides: use of backbone modification to improve specificity

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7 January 2021

Integrated Safety Assessment of 2'-O-Methoxyethyl Chimeric Antisense Oligonucleotides in NonHuman Primates and Healthy Human Volunteers

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7 January 2021

10.1016/S1474-4422(17)30280-6

Identifiers

DOI

1 reference

13 June 2018

PMCID

5604739

1 reference

13 June 2018

PubMed ID

28920889

1 reference

13 June 2018