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AAV9 supports wide-scale transduction of the CNS and TDP-43 disease modeling in adult rats

1 Jan 2015

Abstract excerpt

AAV9 has emerged as an efficient adeno-associated virus (AAV) serotype for gene transfer to the central nervous system. We have used this technique to study aspects of amyotrophic lateral sclerosis (ALS) by administering AAV encoding the ALS-related gene transactive response DNA binding protein of 43 kDa (TDP-43) to neonatal rats. However, inducing the expression in adult subjects would be preferable to mimic the...

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PMID: 26445725
DOI: 10.1038/mtm.2015.36

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