Antisense oligonucleotide therapeutics in neurodegenerative diseases: the case of polyglutamine disorders
Brain : a Journal of Neurology
Nov 18, 2019
Ana Catarina SilvaLuís Pereira de Almeida
PMID: 31738395
DOI: 10.1093/brain/awz328
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Abstract
Polyglutamine (polyQ) disorders are a group of nine neurodegenerative diseases that share a common genetic cause, which is an expansion of CAG repeats in the coding region of the causative genes that are otherwise unrelated. The trinucleotide expansion encodes for an expanded polyQ trac...read more
Mentioned in this Paper
Antistreptolysin O
Cell Growth
Clinical Trials
DNA, Single-Stranded
Genes
Huntington Disease
Nerve Degeneration
Nucleic Acids
RNA Splicing
Chorionic alpha(2)-microglobulin
17
1
Paper Details
References
- References166
- Citations
Pharmacokinetics, biodistribution, and stability of oligodeoxynucleotide phosphorothioates in mice
Proceedings of the National Academy of Sciences of the United States of AmericaSeptember 1, 1991Sudhir AgrawalJ Y Tang41
Polyglutamine expansion as a pathological epitope in Huntington's disease and four dominant cerebellar ataxias
NatureNovember 23, 1995Yvon TrottierLászló Tora
Huntington's disease: from molecular pathogenesis to clinical treatment
Lancet NeurologyJanuary 1, 2011Christopher A Ross, Sarah J Tabrizi3113891
Moderate expansion of a normally biallelic trinucleotide repeat in spinocerebellar ataxia type 2
Nature GeneticsNovember 1, 1996S M PulstS Sahba
Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
Nature MedicineFebruary 1, 2006Julia AlterQi Long Lu1681
Preventing formation of toxic N-terminal huntingtin fragments through antisense oligonucleotide-mediated protein modification
Nucleic Acid TherapeuticsFebruary 1, 2014Melvin M EversWilleke M.C. van Roon-Mom4662
Polyglutamine Repeats in Neurodegenerative Diseases
Annual Review of PathologyAugust 9, 2018Andrew P LiebermanRoger L Albin129
Fully modified 2' MOE oligonucleotides redirect polyadenylation
Nucleic Acids ResearchMarch 15, 2001Timothy A VickersS M FreierIntracellular delivery of an anionic antisense oligonucleotide via receptor-mediated endocytosis
Nucleic Acids ResearchMay 1, 2008Md Rowshon AlamRudolph L Juliano79
Cellular uptake and trafficking of antisense oligonucleotides
Nature BiotechnologyMarch 1, 2017Stanley T CrookeXue-Hai Liang5649313
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- References166
- Citations
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Antisense oligonucleotide therapeutics in neurodegenerative diseases: the case of polyglutamine disorders
Brain : a Journal of Neurology
Nov 18, 2019
Ana Catarina SilvaLuís Pereira de Almeida
PMID: 31738395
DOI: 10.1093/brain/awz328
Abstract
Polyglutamine (polyQ) disorders are a group of nine neurodegenerative diseases that share a common genetic cause, which is an expansion of CAG repeats in the coding region of the causative genes that are otherwise unrelated. The trinucleotide expansion encodes for an expanded polyQ trac...read more
Mentioned in this Paper
Antistreptolysin O
Cell Growth
Clinical Trials
DNA, Single-Stranded
Genes
17
1
Paper Details
References
- References166
- Citations
Pharmacokinetics, biodistribution, and stability of oligodeoxynucleotide phosphorothioates in mice
Proceedings of the National Academy of Sciences of the United States of AmericaSeptember 1, 1991Sudhir AgrawalJ Y Tang41Polyglutamine expansion as a pathological epitope in Huntington's disease and four dominant cerebellar ataxias
NatureNovember 23, 1995Yvon TrottierLászló ToraHuntington's disease: from molecular pathogenesis to clinical treatment
Lancet NeurologyJanuary 1, 2011Christopher A Ross, Sarah J Tabrizi3113891Moderate expansion of a normally biallelic trinucleotide repeat in spinocerebellar ataxia type 2
Nature GeneticsNovember 1, 1996S M PulstS SahbaSystemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology
Nature MedicineFebruary 1, 2006Julia AlterQi Long Lu1681Preventing formation of toxic N-terminal huntingtin fragments through antisense oligonucleotide-mediated protein modification
Nucleic Acid TherapeuticsFebruary 1, 2014Melvin M EversWilleke M.C. van Roon-Mom4662Polyglutamine Repeats in Neurodegenerative Diseases
Annual Review of PathologyAugust 9, 2018Andrew P LiebermanRoger L Albin129Fully modified 2' MOE oligonucleotides redirect polyadenylation
Nucleic Acids ResearchMarch 15, 2001Timothy A VickersS M FreierIntracellular delivery of an anionic antisense oligonucleotide via receptor-mediated endocytosis
Nucleic Acids ResearchMay 1, 2008Md Rowshon AlamRudolph L Juliano79Cellular uptake and trafficking of antisense oligonucleotides
Nature BiotechnologyMarch 1, 2017Stanley T CrookeXue-Hai Liang5649313
12345...
- References166
- Citations
No citations available
This paper may not have been cited yet.
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