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Foreseen accomplishments through precision cellular and molecular biology

dc.contributor.authorAmaral, Olga
dc.contributor.authorDuarte, A.J.
dc.date.accessioned2017-02-21T16:14:47Z
dc.date.available2017-02-21T16:14:47Z
dc.date.issued2016-07
dc.descriptionLecture by Olga Amaral - invitation by the organizing committee.pt_PT
dc.description.abstractLSDs therapies generate large expenses, having a major effect on health systems and family resources. It is, therefore inevitable to attempt to develop effective and economically viable models and therapeutic approaches. Through the development of specific cellular models, we hope to contribute to the increase of choices in terms of therapeutic interventions. Generation and application of iPS cells for the establishment of cellular models for lysosomal diseases has been applied to a few disorders. This approach can allow easier access to disease-specific cells, which will retain the original genotype. We expect that its application to LSDs will provide the means to study pathogenic mechanisms and therapeutic options.pt_PT
dc.description.sponsorshipFederation of Rare Diseases Patients in Central and Eastern Europe; FCT (PTDC/BIM-MEC/4762/2014) 'Modelos celulares para o estudo de mecanismos de disfunção e correção lisossomal'.pt_PT
dc.description.versionN/Apt_PT
dc.identifier.citationOlga Amaral; Foreseen accomplishments through precision cellular and molecular biology, 14th international onference on rare diseases "rare diseases: our task", July 2016, Bialobrzegi-Polóniapt_PT
dc.identifier.urihttp://hdl.handle.net/10400.18/4342
dc.language.isoengpt_PT
dc.peerreviewedyespt_PT
dc.subjectDoenças Genéticaspt_PT
dc.subjectLysosomal Diseasespt_PT
dc.subjectRare Diseasespt_PT
dc.subjectMolecular Therapiespt_PT
dc.subjectCell Modelspt_PT
dc.subjectiPSCspt_PT
dc.titleForeseen accomplishments through precision cellular and molecular biologypt_PT
dc.title.alternativePrzewidywane osiągnięcia dzięki precyzyjnej biologii komórkowej i molekularnejpt_PT
dc.typeconference object
dspace.entity.typePublication
oaire.awardURIinfo:eu-repo/grantAgreement/FCT/3599-PPCDT/PTDC%2FBIM-MEC%2F4762%2F2014/PT
oaire.citation.conferencePlaceBialobrzegi, Polóniapt_PT
oaire.citation.title14th international conference on rare diseases "rare diseases: our task", Federation of rare disease patients in Central and Eastern Europe, 1-3 July 2016pt_PT
oaire.fundingStream3599-PPCDT
project.funder.identifierhttp://doi.org/10.13039/501100001871
project.funder.nameFundação para a Ciência e a Tecnologia
rcaap.rightsrestrictedAccesspt_PT
rcaap.typeconferenceObjectpt_PT
relation.isProjectOfPublication9452b84c-9691-47bb-82ba-75ed40cdfa04
relation.isProjectOfPublication.latestForDiscovery9452b84c-9691-47bb-82ba-75ed40cdfa04

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