Utilize este identificador para referenciar este registo: http://hdl.handle.net/10400.18/4342
Título: Foreseen accomplishments through precision cellular and molecular biology
Outros títulos: Przewidywane osiągnięcia dzięki precyzyjnej biologii komórkowej i molekularnej
Autor: Amaral, Olga
Duarte, A.J.
Palavras-chave: Doenças Genéticas
Lysosomal Diseases
Rare Diseases
Molecular Therapies
Cell Models
iPSCs
Data: Jul-2016
Citação: Olga Amaral; Foreseen accomplishments through precision cellular and molecular biology, 14th international onference on rare diseases "rare diseases: our task", July 2016, Bialobrzegi-Polónia
Resumo: LSDs 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.
Descrição: Lecture by Olga Amaral - invitation by the organizing committee.
Peer review: yes
URI: http://hdl.handle.net/10400.18/4342
Aparece nas colecções:DGH - Apresentações orais em encontros internacionais

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