Gain of toxic function by long-term AAV9-mediated SMN overexpression in the sensorimotor circuit
Gain of toxic function by long-term AAV9-mediated SMN overexpression in the sensorimotor circuit
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New York: Nature Publishing Group US
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English
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New York: Nature Publishing Group US
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The neurodegenerative disease spinal muscular atrophy (SMA) is caused by deficiency in the survival motor neuron (SMN) protein. Currently approved SMA treatments aim to restore SMN, but the potential for SMN expression beyond physiological levels is a unique feature of adeno-associated virus serotype 9 (AAV9)-SMN gene therapy. Here, we show that lo...
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Gain of toxic function by long-term AAV9-mediated SMN overexpression in the sensorimotor circuit
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TN_cdi_proquest_journals_2547168222
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_journals_2547168222
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ISSN
1097-6256
E-ISSN
1546-1726
DOI
10.1038/s41593-021-00827-3
