{"id":50136,"date":"2017-03-14T00:00:00","date_gmt":"2017-03-13T23:00:00","guid":{"rendered":"http:\/\/www.pcb.ub.edu\/new-research-front-to-tackle-friedreichs-ataxia\/"},"modified":"2020-11-18T15:19:37","modified_gmt":"2020-11-18T14:19:37","slug":"new-research-front-to-tackle-friedreichs-ataxia","status":"publish","type":"post","link":"https:\/\/www.pcb.ub.edu\/en\/new-research-front-to-tackle-friedreichs-ataxia\/","title":{"rendered":"New research front to tackle Friedreich\u2019s Ataxia"},"content":{"rendered":"<p>The study is headed by\u00a0Ernest Giralt, head of the\u00a0<a href=\"https:\/\/www.irbbarcelona.org\/en\/research\/design-synthesis-and-structure-of-peptides-and-proteins\" target=\"_blank\" rel=\"noopener noreferrer\">Peptides and Proteins Lab<\/a>, who has many years of experience and is a recognised expert in peptide chemistry and new systems of through which to delivery drugs to the brain, such as peptide shuttles\u2014molecules that have the capacity to carry the drug across the barrier that surrounds and protects the brain. Since the lab started its relation with these patients\u2019 associations in\u00a02013, it has been developing another two projects into Friedrich\u2019s Ataxia.\u00a0In addition to\u00a0Ernest Giralt, the team comprises the scientists\u00a0Meritxell Teixid\u00f3\u00a0and\u00a0Macarena S\u00e1nchez.\u00a0<\/p>\n<p>The long-term goal of the project is to develop a frataxin therapy \u2013ike the injection of insulin for diabetics\u2013\u00a0that reaches the brain\u2014,\u00a0where the reduction of this protein causes the most damage in patients with Friedreich\u2019s Ataxia.\u00a0 \u201cOur peptide shuttles can cross the blood-brain barrier, they reach the central nervous system, and they have not showed signs of toxicity in preliminary studies to date. The challenge in the coming months is to attach frataxin to the shuttles in order to transport the drug across the blood-brain barrier and to test their capacity in cell models,\u201d explains\u00a0Meritxell Teixid\u00f3, Associate Researcher at IRB Barcelona and head of this research line.<\/p>\n<p>\n<strong>A hereditary degenerative disease\u00a0<\/strong><\/p>\n<p>Friedreich\u2019s Ataxia is a hereditary degenerative disease that causes progressive lesions in the nervous system, impairing the coordination of movement and causing muscle weakness, speech problems, and heart conditions. This disease normally appears between the ages of 5 and 25.<\/p>\n<p>\u201cUnfortunately, there are no efficient treatments for the central nervous system for this disease or for others such as Alzheimer\u2019s and Parkinson\u2019s. Getting drugs into the brain continues to be a challenge, but we are hopeful that a cure will eventually be discovered. We believe that the peptide shuttles developed by researchers at IRB Barcelona have enormous potential, although we are aware of the many obstacles to overcome along the way. We consider these molecules to be a good starting point,\u201d say\u00a0Mari Luz Gonz\u00e1lez, and\u00a0Teresa Gilabert, spokespeople of the associations\u00a0Babel Family\u00a0and ASOGAF, respectively.<\/p>\n<p>Two people in every 100,000 are affected by Friedreich\u2019s Ataxia, and this disease hits only those of European origin (Caucasians).\u00a0Spain registers a higher incidence, with an estimated 4.6 cases per 100,000 population. The disease is caused by a defect in a gene, namely frataxin, which results in reduced levels of this protein in the body, particularly affecting the brain, spinal cord and muscles.<br \/>\n\u00a0<\/p>\n<p><strong>\u2022 For further information:\u00a0<a href=\"https:\/\/www.irbbarcelona.org\/en\/news\/new-research-front-to-tackle-friedreichs-ataxia\" target=\"_blank\" rel=\"noopener noreferrer\">IRB Barcelona<\/a><a href=\"https:\/\/www.irbbarcelona.org\/ca\/news\/nou-front-datac-en-la-investigacio-de-lataxia-de-friedreich\" target=\"_blank\" rel=\"noopener noreferrer\"> <\/a><a href=\"https:\/\/www.irbbarcelona.org\/en\/news\/new-research-front-to-tackle-friedreichs-ataxia\" target=\"_blank\" rel=\"noopener noreferrer\">website[+]<\/a><\/strong><\/p>\n<p>\u00a0<\/p>\n","protected":false},"excerpt":{"rendered":"<p>The associations of patients and families\u00a0<a href=\"http:\/\/www.babelfamily.org\/es\/\" target=\"_blank\" rel=\"noopener noreferrer\">Babel Family<\/a>\u00a0and the\u00a0Asociaci\u00f3n Granadina de la Ataxia de Friedreich (<a href=\"http:\/\/www.asogaf.es\/\" target=\"_blank\" rel=\"noopener noreferrer\">ASOGAF<\/a>)\u00a0channel 80,000 euros of their donations (50% from each organisation) into a new 18-month project at the Institute for Research in Biomedicine (<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_000001&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%3Dirb%26_cercador_WAR_empleatsempresesportlet_tab%3D1\" target=\"_blank\" rel=\"noopener noreferrer\">IRB Barcelona<\/a>) located at PCB.\u00a0The long-term goal of the project is to\u00a0achieve an injectable frataxin treatment able to reach the brain.\u00a0Frataxin is the protein that is reduced in those affected by this rare and degenerative disease, which has no cure.<\/p>\n<p>\u00a0<\/p>\n","protected":false},"author":1,"featured_media":55482,"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-50136","post","type-post","status-publish","format-standard","has-post-thumbnail","category-uncategorized"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.0 - 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