Nucleic Acid-Based Therapy Approaches for Huntington's Disease
Huntington's disease (HD) is caused by a dominant mutation that results in an unstable expansion of a CAG repeat in the huntingtin gene leading to a toxic gain of function in huntingtin protein which causes massive neurodegeneration mainly in the striatum and clinical symptoms associated with t...
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| Format: | Article |
| Language: | English |
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Wiley
2012-01-01
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| Series: | Neurology Research International |
| Online Access: | http://dx.doi.org/10.1155/2012/358370 |
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| _version_ | 1832554912131579904 |
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| author | Tatyana Vagner Deborah Young Alexandre Mouravlev |
| author_facet | Tatyana Vagner Deborah Young Alexandre Mouravlev |
| author_sort | Tatyana Vagner |
| collection | DOAJ |
| description | Huntington's disease (HD) is caused by a dominant mutation that results in an unstable expansion of a CAG repeat in the huntingtin gene leading to a toxic gain of function in huntingtin protein which causes massive neurodegeneration mainly in the striatum and clinical symptoms associated with the disease. Since the mutation has multiple effects in the cell and the precise mechanism of the disease remains to be elucidated, gene therapy approaches have been developed that intervene in different aspects of the condition. These approaches include increasing expression of growth factors, decreasing levels of mutant huntingtin, and restoring cell metabolism and transcriptional balance. The aim of this paper is to outline the nucleic acid-based therapeutic strategies that have been tested to date. |
| format | Article |
| id | doaj-art-7c64d853a90e4f039ade94a1b35416f3 |
| institution | Kabale University |
| issn | 2090-1852 2090-1860 |
| language | English |
| publishDate | 2012-01-01 |
| publisher | Wiley |
| record_format | Article |
| series | Neurology Research International |
| spelling | doaj-art-7c64d853a90e4f039ade94a1b35416f32025-02-03T05:50:14ZengWileyNeurology Research International2090-18522090-18602012-01-01201210.1155/2012/358370358370Nucleic Acid-Based Therapy Approaches for Huntington's DiseaseTatyana Vagner0Deborah Young1Alexandre Mouravlev2Department of Molecular Medicine and Pathology and Centre for Brain Research, The University of Auckland, Auckland 1142, New ZealandDepartment of Molecular Medicine and Pathology and Centre for Brain Research, The University of Auckland, Auckland 1142, New ZealandDepartment of Pharmacology and Clinical Pharmacology and Centre for Brain Research, The University of Auckland, Auckland 1142, New ZealandHuntington's disease (HD) is caused by a dominant mutation that results in an unstable expansion of a CAG repeat in the huntingtin gene leading to a toxic gain of function in huntingtin protein which causes massive neurodegeneration mainly in the striatum and clinical symptoms associated with the disease. Since the mutation has multiple effects in the cell and the precise mechanism of the disease remains to be elucidated, gene therapy approaches have been developed that intervene in different aspects of the condition. These approaches include increasing expression of growth factors, decreasing levels of mutant huntingtin, and restoring cell metabolism and transcriptional balance. The aim of this paper is to outline the nucleic acid-based therapeutic strategies that have been tested to date.http://dx.doi.org/10.1155/2012/358370 |
| spellingShingle | Tatyana Vagner Deborah Young Alexandre Mouravlev Nucleic Acid-Based Therapy Approaches for Huntington's Disease Neurology Research International |
| title | Nucleic Acid-Based Therapy Approaches for Huntington's Disease |
| title_full | Nucleic Acid-Based Therapy Approaches for Huntington's Disease |
| title_fullStr | Nucleic Acid-Based Therapy Approaches for Huntington's Disease |
| title_full_unstemmed | Nucleic Acid-Based Therapy Approaches for Huntington's Disease |
| title_short | Nucleic Acid-Based Therapy Approaches for Huntington's Disease |
| title_sort | nucleic acid based therapy approaches for huntington s disease |
| url | http://dx.doi.org/10.1155/2012/358370 |
| work_keys_str_mv | AT tatyanavagner nucleicacidbasedtherapyapproachesforhuntingtonsdisease AT deborahyoung nucleicacidbasedtherapyapproachesforhuntingtonsdisease AT alexandremouravlev nucleicacidbasedtherapyapproachesforhuntingtonsdisease |