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Terapia Génica na Diabetes Mellitus tipo 1

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Detalhes bibliográficos
Resumo:The theme of my monography is "Type 1 Diabetes Mellitus Gene Therapy". Actually, the Diabetes Mellitus is a metabolic disease with a high prevalence worldwide, whose number of cases increases every year. Type I Diabetes Mellitus is one of the existing types of diabetes, in most cases an autoimmune disease resulting from the destruction of pancreatic β cells and as a consequence associated with a complete deficit of endogenous insulin. Presently, the conventional therapeutic treatment of type I Diabetes Mellitus involves administration of recombinant insulin, that promotes a control of glucose levels, but still prone to micro and macrovascular complications associated with hyperglycemia and risks associated with hypoglycemia. These complications affect the quality of life of patients, and therefore new therapeutic alternatives are desirable to improve the current situation. In this context, gene therapy emerged has a potential solution for this disorder. Gene Therapy uses nucleic acids such as DNA or RNA to produce therapeutic effects. Within the scope of type I Diabetes Mellitus, gene therapy can modulate the immune system or reinstate the levels of insulin, through the induction of regeneration of pancreatic β cells or through the production of insulin in non β cells, in particulary hepatocytes. Over this systematic revision, the main strategies of gene therapy applied to type I Diabetes Mellitus will be contextualized, explaining the advantages, the limitations and the future perspectives.
Autores principais:Branco, Marta Salvado Abreu Castelo
Assunto:Diabetes Mellitus tipo 1 Terapia Génica Células β pancreáticas Transdiferenciação Diferenciação Type I Diabetes Mellitus Gene Therapy Pancreatic β cells Transdifferentiation Differentiation
Ano:2019
País:Portugal
Tipo de documento:dissertação de mestrado
Tipo de acesso:acesso aberto
Instituição associada:Universidade de Coimbra
Idioma:português
Origem:Estudo Geral - Universidade de Coimbra
Descrição
Resumo:The theme of my monography is "Type 1 Diabetes Mellitus Gene Therapy". Actually, the Diabetes Mellitus is a metabolic disease with a high prevalence worldwide, whose number of cases increases every year. Type I Diabetes Mellitus is one of the existing types of diabetes, in most cases an autoimmune disease resulting from the destruction of pancreatic β cells and as a consequence associated with a complete deficit of endogenous insulin. Presently, the conventional therapeutic treatment of type I Diabetes Mellitus involves administration of recombinant insulin, that promotes a control of glucose levels, but still prone to micro and macrovascular complications associated with hyperglycemia and risks associated with hypoglycemia. These complications affect the quality of life of patients, and therefore new therapeutic alternatives are desirable to improve the current situation. In this context, gene therapy emerged has a potential solution for this disorder. Gene Therapy uses nucleic acids such as DNA or RNA to produce therapeutic effects. Within the scope of type I Diabetes Mellitus, gene therapy can modulate the immune system or reinstate the levels of insulin, through the induction of regeneration of pancreatic β cells or through the production of insulin in non β cells, in particulary hepatocytes. Over this systematic revision, the main strategies of gene therapy applied to type I Diabetes Mellitus will be contextualized, explaining the advantages, the limitations and the future perspectives.