Martin Marsala
4
Publications
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Gene W Yeo
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Oleksandr Platoshyn
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Sandrine Da Cruz
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Clotilde Lagier-Tourenne
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Melissa McAlonis-Downes
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Christopher E Shaw
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Debbie Swing
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Lino Tessarollo
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Don W Cleveland
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Shuo-Chien Ling
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Philippe A Parone
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Ranjan Batra
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James D Thomas
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Jer-Cherng Chang
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Stefan Aigner
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Anh Bui
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Publications
The sustained expression of Cas9 targeting toxic RNAs reverses disease phenotypes in mouse models of myotonic dystrophy type 1.
Nature biomedical engineering · 2021
ALS-linked TDP-43 mutations produce aberrant RNA splicing and adult-onset motor neuron disease without aggregation or loss of nuclear TDP-43.
Proceedings of the National Academy of Sciences of the United States of America · 2013