{"id":20386,"date":"2020-07-02T16:25:00","date_gmt":"2020-07-02T14:25:00","guid":{"rendered":"https:\/\/ela-international.preprod.melisagency.fr\/?post_type=news_research&p=20386"},"modified":"2024-07-11T17:18:33","modified_gmt":"2024-07-11T15:18:33","slug":"families-researchers-meeting-2020","status":"publish","type":"news_research","link":"https:\/\/elainternational.eu\/en\/actualites_recherche\/families-researchers-meeting-2020\/","title":{"rendered":"Families\/Researchers meeting\u00a02020"},"content":{"rendered":"\n\n
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Families\/Researchers meeting\u00a02020<\/strong><\/h3>\n <\/div>\n
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In view of the exceptional health circumstances, ELA decided not to organise the Families\/Researchers meeting<\/strong>. However, some researchers wanted to submit a summary of their presentation in this ELA Info special n\u00b0110.<\/p>\n <\/div>\n <\/div>\n<\/section>\n\n\n\n

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Summary ELA Info special n\u00b0110
\n<\/strong>Vera Gallo (Italy)\u00a0: Gene therapy of hematopoietic stem cells with a lentiviral vector for metachromatic leukodystrophy, clinical results in 33 patients.
\nRau\u0301l Este\u0301vez (Spain)\u00a0: Reversibility in leukoencephalopathy megalencephalic: molecular and therapeutic mechanisms.
\nDavid A Wenger (United-States)\u00a0: We are getting closer to better treatment of Krabbe disease.
\nElise YAZBECK (France): Minoryx study in France.
\nVivi Heine (The Netherlands)\u00a0: Modeling of 4H syndrome using patient stem cells.
\nFranc\u0327oise Piguet (France)\u00a0: Genetic diseases – workshop Leukodystrophies undetermined.<\/p>\n

Funding Opportunity Announcement, the topics:<\/strong>
\n– Clinical trial\/ preparation for clinical trials in leukodystrophies, including development of long-term effects indicators, characterization of biomarkers, imaging protocols, pharmacodynamic\/ pharmacokinetic studies.
\n– Pre-clinical studies specifically testing therapies (gene, cell, enzymologies or pharmacological therapies) in leukodystrophies.
\n– Development of animal or cellular models (for example iPSC derived from patients) relevant to human leukodystrophies.
\n– Studies of the mechanisms responsible for leukodystrophies, in order to identify new therapeutic approaches.<\/p>\n\n <\/div>\n<\/div>\n\n\n\n\n\n

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ELA Infos<\/strong> special n\u00b0 110<\/h3>\n <\/div>\n
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Download the associated documents<\/h3>\n