Spinal subpial delivery of AAV9 enables widespread gene silencing and blocks motoneuron degeneration in ALS
Author:
Publisher
Springer Science and Business Media LLC
Subject
General Biochemistry, Genetics and Molecular Biology,General Medicine
Link
http://www.nature.com/articles/s41591-019-0674-1.pdf
Reference38 articles.
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2. Matsumoto, A. et al. Disease progression of human SOD1 (G93A) transgenic ALS model rats. J. Neurosci. Res. 83, 119–133 (2006).
3. Kaur, S. J., McKeown, S. R. & Rashid, S. Mutant SOD1 mediated pathogenesis of amyotrophic lateral sclerosis. Gene 577, 109–118 (2016).
4. van Zundert, B. & Brown, R. H. Jr. Silencing strategies for therapy of SOD1-mediated ALS. Neurosci. Lett. 636, 32–39 (2017).
5. Howland, D. S. et al. Focal loss of the glutamate transporter EAAT2 in a transgenic rat model of SOD1 mutant-mediated amyotrophic lateral sclerosis (ALS). Proc. Natl Acad. Sci. USA 99, 1604–1609 (2002).
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