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Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice
Open accessAbstract
Huntington's disease (HD) is a fatal neurodegenerative disorder caused by the presence of an abnormally expanded polyglutamine domain in the N-terminus of huntingtin. We developed a recombinant adeno-associated viral serotype 5 (rAAV5) gene transfer strategy to posttranscriptionally suppress the levels of striatal mutant huntingtin (mHtt) in the R6/1 HD transgenic mouse via RNA interference. Transient cotransfection of HEK293 cells with plasmids expressing a portion of human mHtt derived from R6/1 transgenic HD mice and a short-hairpin RNA directed against the 5' UTR of the mHtt mRNA (siHUNT-1) resulted in reduction in the levels of mHtt mRNA (-75%) and protein (-60%). Long-term in vivo rAAV5-mediated expression of siHUNT-1 in the striatum of R6/1 mice reduced the levels of mHtt mRNA (-78%) and protein (-28%) as determined by quantitative RT-PCR and Western blot analysis, respectively. The reduction in mHtt was concomitant with a reduction in the size and number of neuronal intranuclear inclusions and a small but significant normalization of the steady-state levels of preproenkephalin and dopamine- and cAMP-responsive phosphoprotein 32 kDa mRNA. Finally, bilateral expression of rAAV5-siHUNT-1 resulted in delayed onset of the rear paw clasping phenotype exhibited by the R6/1 mice. These results suggest that a reduction in the levels of striatal mHtt can ameliorate the HD phenotype of R6/1 mice.
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Metadata
- Authors
- Rodriguez-Lebron, E., Denovan-Wright, E. M., Nash, K., Lewin, A. S., & Mandel, R. J.
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2005-10
- Volume
- 12
- Issue
- 4
- Pages
- 618-633
- ISSN
- 1525-0016 (ISSN-L)
- OpenAlex
- W2004817863
- Cited by
- 275
- References
- 70
- FWCI
- 6.11
- Type
- journal article
- Language
- en
- Open access
- hybrid
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- Rodriguez-Lebron, E., Denovan-Wright, E. M., Nash, K., Lewin, A. S., & Mandel, R. J. (2005). Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice. Molecular Therapy. https://doi.org/10.1016/j.ymthe.2005.05.006
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