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Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat

Biochemistry

Oct 30, 2010

Keith T GagnonDavid R Corey

Abstract

Huntington's disease (HD) is a currently incurable neurodegenerative disease caused by the expansion of a CAG trinucleotide repeat within the huntingtin (HTT) gene. Therapeutic approaches include selectively inhibiting the expression of the mutated HTT allele while conserving function o...read more

Mentioned in this Paper

HTT gene
Huntington Disease
Metabolic Inhibition
Phosphorothioate Oligonucleotides
Transcript
Specimen Type - Fibroblasts
Mutation Abnormality
HD protein, human
Nerve Degeneration
Nerve Tissue Proteins
92
1
Paper Details
References
  • References67
  • Citations74
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  • References67
  • Citations74
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Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat

Biochemistry

Oct 30, 2010

Keith T GagnonDavid R Corey

PMID: 21028906

DOI: 10.1021/bi101208k

Abstract

Huntington's disease (HD) is a currently incurable neurodegenerative disease caused by the expansion of a CAG trinucleotide repeat within the huntingtin (HTT) gene. Therapeutic approaches include selectively inhibiting the expression of the mutated HTT allele while conserving function o...read more

Mentioned in this Paper

HTT gene
Huntington Disease
Metabolic Inhibition
Phosphorothioate Oligonucleotides
Transcript
Specimen Type - Fibroblasts
Mutation Abnormality
HD protein, human
Nerve Degeneration
Nerve Tissue Proteins
92
1

Similar Papers Found In These Feeds

Related Papers

Paper Details
References
  • References67
  • Citations74
12345...
  • References67
  • Citations74
12345...
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