Broad distribution of ataxin 1 silencing in rhesus cerebella for spinocerebellar ataxia type 1 therapy (Q34498957)

2 August 2017

Broad distribution of ataxin 1 silencing in rhesus cerebella for spinocerebellar ataxia type 1 therapy (English)

1 reference

spinocerebellar ataxia type 1

2 August 2017

1 reference

2

Jeffrey H Kordower

1 reference

2 August 2017

Megan S Keiser

1

1 reference

2 August 2017

Pedro Gonzalez-Alegre

series ordinal

3

1 reference

2 August 2017

Beverly L Davidson

series ordinal

4

1 reference

2 August 2017

language of work or name

English

0 references

publication date

21 October 2015

1 reference

2 August 2017

1 reference

2 August 2017

138

1 reference

2 August 2017

Pt 12

1 reference

2 August 2017

3555-3566

1 reference

Silencing of end-joining repair for efficient site-specific gene insertion after TALEN/CRISPR mutagenesis in Aedes aegypti

2 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

Single nucleotide seed modification restores in vivo tolerability of a toxic artificial miRNA sequence in the mouse brain

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

Broad therapeutic benefit after RNAi expression vector delivery to deep cerebellar nuclei: implications for spinocerebellar ataxia type 1 therapy

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

AAV9-mediated expression of a non-self protein in nonhuman primate central nervous system triggers widespread neuroinflammation driven by antigen-presenting cell transduction

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

RNAi or overexpression: alternative therapies for Spinocerebellar Ataxia Type 1

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

Pre-existing anti-adeno-associated virus antibodies as a challenge in AAV gene therapy

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

Clinical features, neurogenetics and neuropathology of the polyglutamine spinocerebellar ataxias type 1, 2, 3, 6 and 7.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

Altered Purkinje cell miRNA expression and SCA1 pathogenesis

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

Merits of combination cortical, subcortical, and cerebellar injections for the treatment of Niemann-Pick disease type A

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

AAV2 gene therapy readministration in three adults with congenital blindness.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

Polyglutamine neurodegeneration: expanded glutamines enhance native functions

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

RNA therapeutics: beyond RNA interference and antisense oligonucleotides

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

In vivo monitoring of recovery from neurodegeneration in conditional transgenic SCA1 mice

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

14-3-3 Binding to ataxin-1(ATXN1) regulates its dephosphorylation at Ser-776 and transport to the nucleus

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

Spinocerebellar ataxia type 1.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

Targeted genome editing across species using ZFNs and TALENs

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

In vivo RNAi-mediated alpha-synuclein silencing induces nigrostriatal degeneration

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

Eight years of clinical improvement in MPTP-lesioned primates after gene therapy with AAV2-hAADC.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

Detection of intact rAAV particles up to 6 years after successful gene transfer in the retina of dogs and primates

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

The 14-3-3 proteins: integrators of diverse signaling cues that impact cell fate and cancer development

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

Pathogenic mechanisms of a polyglutamine-mediated neurodegenerative disease, spinocerebellar ataxia type 1

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

Opposing effects of polyglutamine expansion on native protein complexes contribute to SCA1

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

The role of LANP and ataxin 1 in E4F-mediated transcriptional repression

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

RORalpha-mediated Purkinje cell development determines disease severity in adult SCA1 mice

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

VEGF at the neurovascular interface: therapeutic implications for motor neuron disease

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

The AXH domain of Ataxin-1 mediates neurodegeneration through its interaction with Gfi-1/Senseless proteins

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

siRNA-mediated gene silencing in vitro and in vivo.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

The neuropathology of CAG repeat diseases: review and update of genetic and molecular features

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

Expansion of an unstable trinucleotide CAG repeat in spinocerebellar ataxia type 1.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

Expression analysis of the ataxin-1 protein in tissues from normal and spinocerebellar ataxia type 1 individuals

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4840549

Identification and characterization of the gene causing type 1 spinocerebellar ataxia.

7 July 2018

1 reference

https://api.crossref.org/works/10.1093%2FBRAIN%2FAWV292

Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FBRAIN%2FAWV292

Duplication of Atxn1l suppresses SCA1 neuropathology by decreasing incorporation of polyglutamine-expanded ataxin-1 into native complexes.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FBRAIN%2FAWV292

Nuclear inclusions in spinocerebellar ataxia type 1.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FBRAIN%2FAWV292

ATAXIN-1 interacts with the repressor Capicua in its native complex to cause SCA1 neuropathology

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FBRAIN%2FAWV292

Spinocerebellar ataxia type 1 (SCA1): new pathoanatomical and clinico-pathological insights.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FBRAIN%2FAWV292

Gene profiling links SCA1 pathophysiology to glutamate signaling in Purkinje cells of transgenic mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FBRAIN%2FAWV292

RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FBRAIN%2FAWV292

Recovery from polyglutamine-induced neurodegeneration in conditional SCA1 transgenic mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FBRAIN%2FAWV292

Structural and immunocytochemical features of olivopontocerebellar atrophy caused by the spinocerebellar ataxia type 1 (SCA-1) mutation define a unique phenotype

21 January 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/26490326

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1093/BRAIN/AWV292

1 reference

2 August 2017

1 reference

2 August 2017

1 reference