RNA knockdown as a potential therapeutic strategy in Parkinson's disease
Parkinson's disease is a prevalent progressive degenerative disorder of the elderly. There is a current need for novel therapeutic strategies because the standard levodopa pharmacotherapy is only temporarily efficacious. Recently, there have been some high-profile successful preclinical results...
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| Veröffentlicht in: | Gene therapy 2006-03, Vol.13 (6), p.517-524 |
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| creator | Manfredsson, F P Lewin, A S Mandel, R J |
| description | Parkinson's disease is a prevalent progressive degenerative disorder of the elderly. There is a current need for novel therapeutic strategies because the standard levodopa pharmacotherapy is only temporarily efficacious. Recently, there have been some high-profile successful preclinical results obtained in animal models of neurological disorders using small interfering RNAs delivered by viral vectors. RNA interference can theoretically be applied to Parkinson's disease since over-expression of various proteins is known to kill the dopamine neurons of the substantia nigra in animal models and in familial forms of Parkinson's disease. Potential RNA interfering strategies and caveats are discussed in this review. |
| doi_str_mv | 10.1038/sj.gt.3302669 |
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There is a current need for novel therapeutic strategies because the standard levodopa pharmacotherapy is only temporarily efficacious. Recently, there have been some high-profile successful preclinical results obtained in animal models of neurological disorders using small interfering RNAs delivered by viral vectors. RNA interference can theoretically be applied to Parkinson's disease since over-expression of various proteins is known to kill the dopamine neurons of the substantia nigra in animal models and in familial forms of Parkinson's disease. 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There is a current need for novel therapeutic strategies because the standard levodopa pharmacotherapy is only temporarily efficacious. Recently, there have been some high-profile successful preclinical results obtained in animal models of neurological disorders using small interfering RNAs delivered by viral vectors. RNA interference can theoretically be applied to Parkinson's disease since over-expression of various proteins is known to kill the dopamine neurons of the substantia nigra in animal models and in familial forms of Parkinson's disease. Potential RNA interfering strategies and caveats are discussed in this review.</description><subject>Animal models</subject><subject>Animals</subject><subject>Biomedical and Life Sciences</subject><subject>Biomedicine</subject><subject>Care and treatment</subject><subject>Cell Biology</subject><subject>Cell Death - genetics</subject><subject>Diagnosis</subject><subject>Drug therapy</subject><subject>Gene Expression</subject><subject>Gene Expression Regulation</subject><subject>Gene Therapy</subject><subject>Genetic aspects</subject><subject>Genetic Therapy - methods</subject><subject>Health aspects</subject><subject>Human Genetics</subject><subject>Humans</subject><subject>Levodopa</subject><subject>Movement disorders</subject><subject>Nanotechnology</subject><subject>Neurodegenerative diseases</subject><subject>Neurological diseases</subject><subject>Overexpression</subject><subject>Parkinson Disease - genetics</subject><subject>Parkinson Disease - therapy</subject><subject>Parkinson's disease</subject><subject>Proteins - genetics</subject><subject>review</subject><subject>Ribonucleic acid</subject><subject>RNA</subject><subject>RNA Interference</subject><subject>RNA, Small Interfering - genetics</subject><subject>RNA-mediated interference</subject><subject>Substantia nigra</subject><subject>Viruses</subject><issn>0969-7128</issn><issn>1476-5462</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2006</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><sourceid>8G5</sourceid><sourceid>BENPR</sourceid><sourceid>GUQSH</sourceid><sourceid>M2O</sourceid><recordid>eNqFkt1rFDEUxQdRbK0--iqDQsWHWfOdmcelqC0UlarPIZu5Mzu7s8mam0H735uli-tKRfIQSH73hHNyiuI5JTNKeP0WV7M-zTgnTKnmQXFKhVaVFIo9LE5Jo5pKU1afFE8QV4QQoWv2uDihiiktNTktLm8-zsu1D27dhh--tFjachsS-DTYsUxLiHYLUxpciSnaBP1tOfjys43rwWPwr7FsBwSL8LR41NkR4dl-Pyu-vX_39eKyuv704epifl05JXmqNGlb5epGcw1AWsJqKYkmDafWiU7VnXLCsYUUrHFMdk5akLAQi84q1nEQ_Kw4v9PdxvB9AkxmM6CDcbQewoRGac0kb_h_QdrUXNaSZfDVX-AqTNFnE4YpIZRgWu3kXv6TorVmXGUTv6V6O4IZfBdyaG73rpnTmmlNeUMzNbuHyquFzeCCh27I50cDb44GMpPgZ-rthGiuvtwcs-d_sEuwY1piGPMXBo_HYHUHuhgQI3RmG4eNjbeGErOrlsGV6ZPZVyvzL_YBTIsNtAd636WDLcxXvod4SOh-xV-w4tRT</recordid><startdate>20060301</startdate><enddate>20060301</enddate><creator>Manfredsson, F P</creator><creator>Lewin, A S</creator><creator>Mandel, R J</creator><general>Nature Publishing Group UK</general><general>Nature Publishing Group</general><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>ISR</scope><scope>3V.</scope><scope>7QP</scope><scope>7TK</scope><scope>7TM</scope><scope>7U9</scope><scope>7X7</scope><scope>7XB</scope><scope>88E</scope><scope>8AO</scope><scope>8C1</scope><scope>8FD</scope><scope>8FE</scope><scope>8FH</scope><scope>8FI</scope><scope>8FJ</scope><scope>8FK</scope><scope>8G5</scope><scope>ABUWG</scope><scope>AFKRA</scope><scope>AZQEC</scope><scope>BBNVY</scope><scope>BENPR</scope><scope>BHPHI</scope><scope>CCPQU</scope><scope>DWQXO</scope><scope>FR3</scope><scope>FYUFA</scope><scope>GHDGH</scope><scope>GNUQQ</scope><scope>GUQSH</scope><scope>H94</scope><scope>HCIFZ</scope><scope>K9.</scope><scope>LK8</scope><scope>M0S</scope><scope>M1P</scope><scope>M2O</scope><scope>M7P</scope><scope>MBDVC</scope><scope>P64</scope><scope>PQEST</scope><scope>PQQKQ</scope><scope>PQUKI</scope><scope>PRINS</scope><scope>Q9U</scope><scope>RC3</scope><scope>7QO</scope><scope>7X8</scope></search><sort><creationdate>20060301</creationdate><title>RNA knockdown as a potential therapeutic strategy in Parkinson's disease</title><author>Manfredsson, F P ; Lewin, A S ; Mandel, R J</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c653t-70dd6c89737ee0d02855070931ac4f68f6c4c2b5429c25fc5ae5eb4bfa62f3e43</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2006</creationdate><topic>Animal models</topic><topic>Animals</topic><topic>Biomedical and Life Sciences</topic><topic>Biomedicine</topic><topic>Care and treatment</topic><topic>Cell Biology</topic><topic>Cell Death - 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| source | MEDLINE; Free E-Journal (出版社公開部分のみ）; Springer Online Journals - JUSTICE |
| subjects | Animal models Animals Biomedical and Life Sciences Biomedicine Care and treatment Cell Biology Cell Death - genetics Diagnosis Drug therapy Gene Expression Gene Expression Regulation Gene Therapy Genetic aspects Genetic Therapy - methods Health aspects Human Genetics Humans Levodopa Movement disorders Nanotechnology Neurodegenerative diseases Neurological diseases Overexpression Parkinson Disease - genetics Parkinson Disease - therapy Parkinson's disease Proteins - genetics review Ribonucleic acid RNA RNA Interference RNA, Small Interfering - genetics RNA-mediated interference Substantia nigra Viruses |
| title | RNA knockdown as a potential therapeutic strategy in Parkinson's disease |
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