Familial cortical dysplasia caused by mutation in the mammalian target of rapamycin regulator NPRL3

© 2015 American Neurological Association. We describe first cousin sibling pairs with focal epilepsy, one of each pair having focal cortical dysplasia (FCD) IIa. Linkage analysis and whole-exome sequencing identified a heterozygous germline frameshift mutation in the gene encoding nitrogen permease...

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Main Authors: Sim, J., Scerri, T., Fanjul-Fernández, M., Riseley, J., Gillies, G., Pope, K., Van Roozendaal, H., Heng, Julian, Mandelstam, S., McGillivray, G., Macgregor, D., Kannan, L., Maixner, W., Harvey, A., Amor, D., Delatycki, M., Crino, P., Bahlo, M., Lockhart, P., Leventer, R.
Format: Journal Article
Published: John Wiley and Son 2016
Online Access:http://hdl.handle.net/20.500.11937/71048
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author Sim, J.
Scerri, T.
Fanjul-Fernández, M.
Riseley, J.
Gillies, G.
Pope, K.
Van Roozendaal, H.
Heng, Julian
Mandelstam, S.
McGillivray, G.
Macgregor, D.
Kannan, L.
Maixner, W.
Harvey, A.
Amor, D.
Delatycki, M.
Crino, P.
Bahlo, M.
Lockhart, P.
Leventer, R.
author_facet Sim, J.
Scerri, T.
Fanjul-Fernández, M.
Riseley, J.
Gillies, G.
Pope, K.
Van Roozendaal, H.
Heng, Julian
Mandelstam, S.
McGillivray, G.
Macgregor, D.
Kannan, L.
Maixner, W.
Harvey, A.
Amor, D.
Delatycki, M.
Crino, P.
Bahlo, M.
Lockhart, P.
Leventer, R.
author_sort Sim, J.
building Curtin Institutional Repository
collection Online Access
description © 2015 American Neurological Association. We describe first cousin sibling pairs with focal epilepsy, one of each pair having focal cortical dysplasia (FCD) IIa. Linkage analysis and whole-exome sequencing identified a heterozygous germline frameshift mutation in the gene encoding nitrogen permease regulator-like 3 (NPRL3). NPRL3 is a component of GAP Activity Towards Rags 1, a negative regulator of the mammalian target of rapamycin complex 1 signaling pathway. Immunostaining of resected brain tissue demonstrated mammalian target of rapamycin activation. Screening of 52 unrelated individuals with FCD identified 2 additional patients with FCDIIa and germline NPRL3 mutations. Similar to DEPDC5, NPRL3 mutations may be considered as causal variants in patients with FCD or magnetic resonance imaging-negative focal epilepsy.
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spelling curtin-20.500.11937-710482018-12-13T09:33:09Z Familial cortical dysplasia caused by mutation in the mammalian target of rapamycin regulator NPRL3 Sim, J. Scerri, T. Fanjul-Fernández, M. Riseley, J. Gillies, G. Pope, K. Van Roozendaal, H. Heng, Julian Mandelstam, S. McGillivray, G. Macgregor, D. Kannan, L. Maixner, W. Harvey, A. Amor, D. Delatycki, M. Crino, P. Bahlo, M. Lockhart, P. Leventer, R. © 2015 American Neurological Association. We describe first cousin sibling pairs with focal epilepsy, one of each pair having focal cortical dysplasia (FCD) IIa. Linkage analysis and whole-exome sequencing identified a heterozygous germline frameshift mutation in the gene encoding nitrogen permease regulator-like 3 (NPRL3). NPRL3 is a component of GAP Activity Towards Rags 1, a negative regulator of the mammalian target of rapamycin complex 1 signaling pathway. Immunostaining of resected brain tissue demonstrated mammalian target of rapamycin activation. Screening of 52 unrelated individuals with FCD identified 2 additional patients with FCDIIa and germline NPRL3 mutations. Similar to DEPDC5, NPRL3 mutations may be considered as causal variants in patients with FCD or magnetic resonance imaging-negative focal epilepsy. 2016 Journal Article http://hdl.handle.net/20.500.11937/71048 10.1002/ana.24502 John Wiley and Son restricted
spellingShingle Sim, J.
Scerri, T.
Fanjul-Fernández, M.
Riseley, J.
Gillies, G.
Pope, K.
Van Roozendaal, H.
Heng, Julian
Mandelstam, S.
McGillivray, G.
Macgregor, D.
Kannan, L.
Maixner, W.
Harvey, A.
Amor, D.
Delatycki, M.
Crino, P.
Bahlo, M.
Lockhart, P.
Leventer, R.
Familial cortical dysplasia caused by mutation in the mammalian target of rapamycin regulator NPRL3
title Familial cortical dysplasia caused by mutation in the mammalian target of rapamycin regulator NPRL3
title_full Familial cortical dysplasia caused by mutation in the mammalian target of rapamycin regulator NPRL3
title_fullStr Familial cortical dysplasia caused by mutation in the mammalian target of rapamycin regulator NPRL3
title_full_unstemmed Familial cortical dysplasia caused by mutation in the mammalian target of rapamycin regulator NPRL3
title_short Familial cortical dysplasia caused by mutation in the mammalian target of rapamycin regulator NPRL3
title_sort familial cortical dysplasia caused by mutation in the mammalian target of rapamycin regulator nprl3
url http://hdl.handle.net/20.500.11937/71048