Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients
about
Gene suppression strategies for dominantly inherited neurodegenerative diseases: lessons from Huntington's disease and spinocerebellar ataxia.Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy.HD iPSC-derived neural progenitors accumulate in culture and are susceptible to BDNF withdrawal due to glutamate toxicity.Comparative analysis of anti-polyglutamine Fab crystals grown on Earth and in microgravity.Gene-targeting pharmaceuticals for single-gene disorders.Targeting Long Noncoding RNA with Antisense Oligonucleotide Technology as Cancer Therapeutics.Huntington's Disease-Update on Treatments.New developments in RAN translation: insights from multiple diseases.Antisense Oligonucleotides: Translation from Mouse Models to Human Neurodegenerative Diseases.Huntingtin Haplotypes Provide Prioritized Target Panels for Allele-specific Silencing in Huntington Disease Patients of European Ancestry.CTG repeat-targeting oligonucleotides for down-regulating Huntingtin expression.Influence of mismatched and bulged nucleotides on SNP-preferential RNase H cleavage of RNA-antisense gapmer heteroduplexes.Gene suppression approaches to neurodegeneration.Optimization of trans-Splicing for Huntington's Disease RNA Therapy.Transfer of genetic therapy across human populations: molecular targets for increasing patient coverage in repeat expansion diseases.A Tandem Oligonucleotide Approach for SNP-Selective RNA Degradation Using Modified Antisense Oligonucleotides.Epidemiology of Huntington disease: first post-HTT gene analysis of prevalence in Italy.HACE1 is essential for astrocyte mitochondrial function and influences Huntington disease phenotypes in vivo.Antisense oligonucleotide therapy for spinocerebellar ataxia type 2.Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs.Use of antisense oligonucleotides to correct the splicing error in ISCU myopathy patient cell lines.RNA-directed off/on switch of RNase H activity using boronic ester formation.Preventing mutant huntingtin proteolysis and intermittent fasting promote autophagy in models of Huntington disease.Therapy development in Huntington disease: From current strategies to emerging opportunities.A human huntingtin SNP alters post-translational modification and pathogenic proteolysis of the protein causing Huntington disease.
P2860
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P2860
Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients
description
2014 nî lūn-bûn
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2014 թուականին հրատարակուած գիտական յօդուած
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2014 թվականին հրատարակված գիտական հոդված
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2014年の論文
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2014年論文
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2014年論文
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2014年論文
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2014年論文
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2014年論文
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2014年论文
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name
Allele-specific suppression of ...... ll Huntington disease patients
@ast
Allele-specific suppression of ...... ll Huntington disease patients
@en
Allele-specific suppression of ...... ll Huntington disease patients
@nl
type
label
Allele-specific suppression of ...... ll Huntington disease patients
@ast
Allele-specific suppression of ...... ll Huntington disease patients
@en
Allele-specific suppression of ...... ll Huntington disease patients
@nl
prefLabel
Allele-specific suppression of ...... ll Huntington disease patients
@ast
Allele-specific suppression of ...... ll Huntington disease patients
@en
Allele-specific suppression of ...... ll Huntington disease patients
@nl
P2093
P2860
P50
P1433
P1476
Allele-specific suppression of ...... ll Huntington disease patients
@en
P2093
Andrew T Watt
C Frank Bennett
Crystal N Doty
Eric E Swayze
Eugenia Petoukhov
Holly Kordasiewicz
Jeffrey B Carroll
Kuljeet Vaid
Michael E Østergaard
P2860
P304
P356
10.1371/JOURNAL.PONE.0107434
P407
P577
2014-01-01T00:00:00Z