Activity and High-Order Effective Connectivity Alterations in Sanfilippo C Patient-Specific Neuronal Networks
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- 作者：Isaac Canals1 ; 2 ; 3 ; Jordi Soriano4 ; Javier G. Orlandi4 ; Roger Torrent3 ; Yvonne Richaud-Patin5 ; 6 ; Senda Jimé ; nez-Delgado5 ; 6 ; Simone Merlin7 ; Antonia Follenzi7 ; Antonella Consiglio3 ; 8 ; Lluï ; sa Vilageliu1 ; 2 ; 3 ; Daniel Grinberg1 ; 2 ; 3 ; dgrinberg@ub.edu" class="auth_mail" title="E-mail the corresponding author ; Angel Raya5 ; 6 ; 9 ; araya@cmrb.eu" class="auth_mail" title="E-mail the corresponding author
- 刊名：Stem Cell Reports
- 出版年：2015
- 出版时间：13 October 2015
- 年：2015
- 卷：5
- 期：4
- 页码：546-557
- 全文大小：2930 K
文摘
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Fibroblasts from two Sanfilippo C patients were reprogrammed to obtain iPSCs
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iPSCs were successfully differentiated to neural cells that mimic the disease
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Networks of patients’ neurons show altered activity and connectivity
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Early functional phenotypes are prevented in gene-corrected patients’ neurons