RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model.

By S. Harper, Patrick D. Staber, Xiaohua He, S. Eliason, Inês H Martins, Q. Mao, Linda C. Yang, R. Kotin, H. Paulson, B. Davidson. Proceedings of the National Academy of Sciences of the United States of America, 2005

694 citations recorded by the source corpus.

Subfields: Cellular and Molecular Neuroscience, Molecular Biology

View Paper: https://www.semanticscholar.org/paper/54417bacd3baf32712dac3ee4d64ac55084b3cde

How researchers see this paper

The researchers whose trusted networks rank it highest, strongest advocate first. Every researcher gets a different order.

In Cellular and Molecular Neuroscience

1. C. Zuccato
2. E. Cattaneo
3. M. Valenza
4. D. Rubinsztein
5. N. Mizushima
6. M. Jäättelä
7. M. Høyer-Hansen

In Molecular Biology

1. P. McCray
2. M. Valenza
3. E. Cattaneo
4. C. Zuccato
5. D. Rubinsztein
6. B. Davidson
7. N. Mizushima
8. M. Jäättelä

Citations in the corpus

• Intravascular AAV9 preferentially targets neonatal-neurons and adult-astrocytes in CNS (2008, background): “It is noteworthy that AAV9 vascular delivery in the adult does not achieve widespread direct neuronal targeting, precluding the use of this approach for diseases such as Huntington's, in which multiple structures and brain regions are likely to require neuronal targeting to suppress mutant Huntingti”
• Current prospects for RNA interference-based therapies (2011, background): “For example, vectors expressing therapeutic RNAi improved disease phenotypes for many months in preclinical studies in rodent models of polyglutamine repeat diseases [108] [109] [110] [111] [112] [113] , amyotrophic lateral sclerosis [114] [115] [116] , Parkinson's disease 117 and Alzheimer's diseas”
• Normal huntingtin function: an alternative approach to Huntington's disease (2005, background): “The reduced physiological activity of huntingtin in HD might also affect the development of therapeutic strategies: for example, RNA INTERFERENCE (RNAi) strategies aimed at blocking the disease-triggering mutation would have to be selective for the mutant allel”
• Molecular mechanisms and potential therapeutical targets in Huntington's disease. (2010, background): “Notably, silencing the exogenously expressed pathogenic gene prevented behavioral and neuropathological symptoms in HD mice, with particular improvements in stride length measurements and rotaroad performance, and also reduction in mutant huntingtin inclusions (243).”
• Sustained therapeutic reversal of Huntington’s disease by transient repression of huntingtin synthesis (2012, neutral): “…needs to be delivered by direct intraparenchymal injections, and therapeutic correction is limited to only a small portion of the striatum immediately adjacent to the sites of injection (Boudreau et al., 2009; DiFiglia et al., 2007; Drouet et al., 2009; Harper et al., 2005; McBride et al., 2008).”
• Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi (2008, methodology): “Procedures were performed as reported previously (8) with the following exceptions.”
• The roles of intracellular protein-degradation pathways in neurodegeneration (2006, background): “Promising progress has been made towards selective synthesis inhibition of dominant gain-of-function mutations, such as the mutant HD allel”