{"id":50298,"date":"2015-10-26T00:00:00","date_gmt":"2015-10-25T23:00:00","guid":{"rendered":"http:\/\/www.pcb.ub.edu\/a-study-applies-ipsc-technology-to-sanfilippo-syndrome-research\/"},"modified":"2015-10-26T00:00:00","modified_gmt":"2015-10-25T23:00:00","slug":"a-study-applies-ipsc-technology-to-sanfilippo-syndrome-research","status":"publish","type":"post","link":"https:\/\/www.pcb.ub.edu\/en\/a-study-applies-ipsc-technology-to-sanfilippo-syndrome-research\/","title":{"rendered":"A study applies iPSC technology to Sanfilippo syndrome research"},"content":{"rendered":"<p>The investigation has generated mature neurons from patient-specific iPSC lines that recapitulate the main characteristics of the disease, those not present in genetically corrected patient-specific iPSC-derived cultures overexpressing the cDNA of the\u00a0HGSNAT\u00a0gene.<\/p>\n<p>Furthermore, neuronal networks organized in vitro from mature patient-derived neurons showed early defects in neuronal activity, network-wide degradation, and altered effective connectivity. Findings establish the importance of iPSC-based technology to identify early functional phenotypes, which can in turn shed light on the pathological mechanisms occurring in Sanfilippo syndrome. The technology can be also useful to analyse potential therapeutic compounds that could prevent the onset of neurodegeneration.<\/p>\n<p>Daniel Grinberg, Isaac Canals and Lu\u00efsa Vilageliu, researchers in the Department of Genetics and members of IBUB\u00a0and the Centre for Biomedical Network Research on Rare Diseases (CIBERER), have collaborated in the study, together with Jordi Soriano and Javier G. Orlandi, experts from the Faculty of Physics, and the research group led by Angel Raya, director of the Center of Regenerative Medicine in Barcelona and researcher at IBEC.<br \/>\n\u00a0<\/p>\n<p><strong>Article reference:<\/strong><\/p>\n<p>Canals I., Soriano J, Orlandi J. G., Torrent R., Richaud-Patin Y., Jim\u00e9nez-Delgado S., Merlin S., Follenzi A., Consiglio A., Vilageliu Ll., Grinberg D., Raya A.\u00a0<a href=\"http:\/\/www.cell.com\/stem-cell-reports\/abstract\/S2213-6711%2815%2900250-7\">\u201cActivity and High-Order Effective Connectivity Alterations in Sanfilippo C Patient-Specific Neuronal Networks\u201d<\/a>.\u00a0Stem Cell Reports, October 2015. DOI: 10.1016\/j.stemcr.2015.08.016<\/p>\n<p>\u00a0<\/p>\n","protected":false},"excerpt":{"rendered":"<p>Sanfilippo type c syndrome is a lysosomal storage disorder caused by mutations in the\u00a0HGSNAT\u00a0gene that leads to progressive neurodegeneration. A study, which has just been published in the journal\u00a0Stem Cell Reports, has generated patient-specific induced pluripotent stem cells (iPSC) which can contribute to know better disease\u2019s pathological mechanisms and design new therapeutic options. A group of researchers from the <a href=\"http:\/\/www.pcb.ub.edu\/portal\/en\/cerca?p_p_id=cercador_WAR_empleatsempresesportlet&amp;p_p_lifecycle=0&amp;p_p_state=maximized&amp;p_p_mode=view&amp;p_p_col_id=column-1&amp;p_p_col_count=1&amp;_cercador_WAR_empleatsempresesportlet_jspPage=%2Fhtml%2Fcommon%2Fdetall-empresa.jsp&amp;_cercador_WAR_empleatsempresesportlet_empresaId=ENT_002730&amp;_cercador_WAR_empleatsempresesportlet_redirect=%2Fportal%2Fes%2Fcerca%3Fp_p_id%3Dcercador_WAR_empleatsempresesportlet%26p_p_lifecycle%3D0%26p_p_mode%3Dview%26_cercador_WAR_empleatsempresesportlet_keywords%3DIBUB%26_cercador_WAR_empleatsempresesportlet_tab%3D1\">Institute of Biomedicine of the University of Barcelona\u00a0<\/a>(<a href=\"http:\/\/www.pcb.ub.edu\/portal\/en\/cerca?p_p_id=cercador_WAR_empleatsempresesportlet&amp;p_p_lifecycle=0&amp;p_p_state=maximized&amp;p_p_mode=view&amp;p_p_col_id=column-1&amp;p_p_col_count=1&amp;_cercador_WAR_empleatsempresesportlet_jspPage=%2Fhtml%2Fcommon%2Fdetall-empresa.jsp&amp;_cercador_WAR_empleatsempresesportlet_empresaId=ENT_002730&amp;_cercador_WAR_empleatsempresesportlet_redirect=%2Fportal%2Fes%2Fcerca%3Fp_p_id%3Dcercador_WAR_empleatsempresesportlet%26p_p_lifecycle%3D0%26p_p_mode%3Dview%26_cercador_WAR_empleatsempresesportlet_keywords%3DIBUB%26_cercador_WAR_empleatsempresesportlet_tab%3D1\" target=\"_blank\" rel=\"noopener noreferrer\">IBUB)\u00a0<\/a>and <a href=\"http:\/\/www.pcb.ub.edu\/portal\/en\/cerca?p_p_id=cercador_WAR_empleatsempresesportlet&amp;p_p_lifecycle=0&amp;p_p_state=maximized&amp;p_p_mode=view&amp;p_p_col_id=column-1&amp;p_p_col_count=1&amp;_cercador_WAR_empleatsempresesportlet_jspPage=%2Fhtml%2Fcommon%2Fdetall-empresa.jsp&amp;_cercador_WAR_empleatsempresesportlet_empresaId=ENT_000134&amp;_cercador_WAR_empleatsempresesportlet_redirect=%2Fportal%2Fca%2Fcerca%3Fp_p_id%3Dcercador_WAR_empleatsempresesportlet%26p_p_lifecycle%3D0%26p_p_mode%3Dview%26_cercador_WAR_empleatsempresesportlet_keywords%3Dibec%26_cercador_WAR_empleatsempresesportlet_tab%3D1\" target=\"_blank\" rel=\"noopener noreferrer\">Institute for Bioengineering of Catalonia (IBEC)<\/a>\u00a0at PCB\u00a0has participated in the study.\u00a0<\/p>\n","protected":false},"author":1,"featured_media":0,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"inline_featured_image":false,"footnotes":""},"categories":[8],"tags":[],"class_list":["post-50298","post","type-post","status-publish","format-standard","category-uncategorized"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.5 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>A study applies iPSC technology to Sanfilippo syndrome research - Parc Cient\u00edfic de Barcelona<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.pcb.ub.edu\/en\/a-study-applies-ipsc-technology-to-sanfilippo-syndrome-research\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"A study applies iPSC technology to Sanfilippo syndrome research - Parc Cient\u00edfic de Barcelona\" \/>\n<meta property=\"og:description\" content=\"Sanfilippo type c syndrome is a lysosomal storage disorder caused by mutations in the\u00a0HGSNAT\u00a0gene that leads to progressive neurodegeneration. A study, which has just been published in the journal\u00a0Stem Cell Reports, has generated patient-specific induced pluripotent stem cells (iPSC) which can contribute to know better disease\u2019s pathological mechanisms and design new therapeutic options. A group of researchers from the Institute of Biomedicine of the University of Barcelona\u00a0(IBUB)\u00a0and Institute for Bioengineering of Catalonia (IBEC)\u00a0at PCB\u00a0has participated in the study.\u00a0\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.pcb.ub.edu\/en\/a-study-applies-ipsc-technology-to-sanfilippo-syndrome-research\/\" \/>\n<meta property=\"og:site_name\" content=\"Parc Cient\u00edfic de Barcelona\" \/>\n<meta property=\"article:published_time\" content=\"2015-10-25T23:00:00+00:00\" \/>\n<meta name=\"author\" content=\"adminpcb\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"adminpcb\" \/>\n\t<meta name=\"twitter:label2\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"1 minute\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\\\/\\\/www.pcb.ub.edu\\\/en\\\/a-study-applies-ipsc-technology-to-sanfilippo-syndrome-research\\\/#article\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/www.pcb.ub.edu\\\/en\\\/a-study-applies-ipsc-technology-to-sanfilippo-syndrome-research\\\/\"},\"author\":{\"name\":\"adminpcb\",\"@id\":\"https:\\\/\\\/www.pcb.ub.edu\\\/es\\\/#\\\/schema\\\/person\\\/d13cab05cd0a75b17fddca8496481021\"},\"headline\":\"A study applies iPSC technology to Sanfilippo syndrome research\",\"datePublished\":\"2015-10-25T23:00:00+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\\\/\\\/www.pcb.ub.edu\\\/en\\\/a-study-applies-ipsc-technology-to-sanfilippo-syndrome-research\\\/\"},\"wordCount\":225,\"inLanguage\":\"en-US\"},{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/www.pcb.ub.edu\\\/en\\\/a-study-applies-ipsc-technology-to-sanfilippo-syndrome-research\\\/\",\"url\":\"https:\\\/\\\/www.pcb.ub.edu\\\/en\\\/a-study-applies-ipsc-technology-to-sanfilippo-syndrome-research\\\/\",\"name\":\"A study applies iPSC technology to Sanfilippo syndrome research - 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