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Authors
Advisor(s)
Abstract(s)
As anomalias congénitas correspondem à quinta principal causa de morte em crianças
com menos de 5 anos, em todo o mundo, representando um total de 532854 mortes em
2019. Constituem, assim, um grave problema de saúde pública a nível mundial, sendo
uma importante causa de mortalidade, mobilidade e hospitalização. Apesar dos avanços
na Medicina, as opções terapêuticas para o tratamento destas malformações são ainda
muito limitadas, sendo necessário encontrar alternativas válidas que permitam melhorar
os outcomes clínicos destas crianças. As células estaminais do líquido amniótico possuem
um conjunto de propriedades e características únicas e incomparáveis que lhes conferem
um enorme potencial terapêutico no campo da Medicina Regenerativa fetal/pediátrica,
nomeadamente no que toca ao tratamento de anomalias congénitas. Recentemente, a
TRASCET – Transamniotic Stem Cell Therapy -, por se basear no papel natural
desempenhado por estas células, tem surgido como uma opção terapêutica promissora
com enorme potencial no tratamento de algumas das anomalias congénitas mais
frequentemente diagnosticadas, parecendo ser uma opção mais acessível, menos invasiva,
custo-efetiva e eficaz.
Esta dissertação pretende então descrever o estado atual da arte no que concerne à
aplicação terapêutica com células estaminais do líquido amniótico no tratamento de
anomalias congénitas – TRASCET -, nomeadamente a sua aplicabilidade em algumas das
anomalias congénitas mais frequentes – Mielomeningocelo, incluindo a malformação de
Chiari II, Gastrosquise e Hérnia Diafragmática Congénita. Foram realizadas pesquisas nas
plataformas PubMed e Google Scholar, tendo por base as palavras-chave “Stem Cells”,
“Amniotic Fluid”, “Congenital Anomalies” e “TRASCET”.
Concluiu-se que, de facto, a TRASCET tem impacto no tratamento destas anomalias
congénitas e a evidência atual suporta a hipótese de que as células estaminais do líquido
amniótico representam uma fonte promissora com enorme potencial terapêutico em
diversas anomalias congénitas. Ainda que a translação dos resultados destes estudos
experimentais para a clínica possa ser uma tarefa complexa, é altamente provável que os
resultados que têm vindo a ser obtidos através de estudos pré-clínicos em modelos
animais sejam transladados para fetos humanos dentro de poucos anos. É imperativo
aprofundar o estudo destas células e da aplicabilidade da TRASCET, quer em estudos préclínicos como clínicos.
Congenital anomalies are the fifth leading cause of death in children under 5 years of age worlwide, representing a total of 532854 deaths in 2019. Thus, they constitute a grave public health problem, being an important cause of mortality, morbidity and hospitalization. Despite all the advances in Medicine, the therapeutic options for the treatment of these malformations are still very limited, and it’s necessary to find valid alternatives that improve the clinical outcomes of these children. Amniotic fluid stem cells have a set of unique and incomparable properties and characteristics that give them an enormous therapeutic potentail in the field of fetal/pediatric Regenerative Medicine, particulary with regard to the treatment of congenital anomalies. Recently, TRASCET – Transamniotic Stem Cell Therapy -, based on the natural role played by these cells, has emerged as a promising therapeutic option with enormous potential in the treatement of some of the most frequently diagnosed congenital anomalies, appearing to be a more accessible, less invasive, cost-effective and eficiente option. This dissertation aims to describe the current state of art regarding the therapeutic aplication with amniotic fluid stem cells in the treatement of congenital anomalies – TRASCET -, namely its applicability in some of the most frequent congenital anomalies – Myelomeningocele, including the Chiari Malformation type II, Gastroschisis and Diaphragmatic Congenital Hernia. Research was carried out on the PubMed and Google Schoolar platforms, based on the keywords “Stem Cells”, “Amniotic Fluid”, “Congenital Anomalies” and “TRASCET”. It was concluded that, in fact, TRASCET has an impact on the treatment of these congenital anomalities and the current evidence supports the hypothesis that amniotic fluid stem cells represent a promising source with enormous therapeutic potential in various congenital anomalies. Although translating the results of these experimental studies to the clinic can be a complex task, it’s highly likely that the results that have been obtained from preclinical studies in animal models will be translated to human fetuses within a few years. It’s imperative to deepen the study of these cells and the applicability of TRASCET, both in pre-clinical and clinical studies.
Congenital anomalies are the fifth leading cause of death in children under 5 years of age worlwide, representing a total of 532854 deaths in 2019. Thus, they constitute a grave public health problem, being an important cause of mortality, morbidity and hospitalization. Despite all the advances in Medicine, the therapeutic options for the treatment of these malformations are still very limited, and it’s necessary to find valid alternatives that improve the clinical outcomes of these children. Amniotic fluid stem cells have a set of unique and incomparable properties and characteristics that give them an enormous therapeutic potentail in the field of fetal/pediatric Regenerative Medicine, particulary with regard to the treatment of congenital anomalies. Recently, TRASCET – Transamniotic Stem Cell Therapy -, based on the natural role played by these cells, has emerged as a promising therapeutic option with enormous potential in the treatement of some of the most frequently diagnosed congenital anomalies, appearing to be a more accessible, less invasive, cost-effective and eficiente option. This dissertation aims to describe the current state of art regarding the therapeutic aplication with amniotic fluid stem cells in the treatement of congenital anomalies – TRASCET -, namely its applicability in some of the most frequent congenital anomalies – Myelomeningocele, including the Chiari Malformation type II, Gastroschisis and Diaphragmatic Congenital Hernia. Research was carried out on the PubMed and Google Schoolar platforms, based on the keywords “Stem Cells”, “Amniotic Fluid”, “Congenital Anomalies” and “TRASCET”. It was concluded that, in fact, TRASCET has an impact on the treatment of these congenital anomalities and the current evidence supports the hypothesis that amniotic fluid stem cells represent a promising source with enormous therapeutic potential in various congenital anomalies. Although translating the results of these experimental studies to the clinic can be a complex task, it’s highly likely that the results that have been obtained from preclinical studies in animal models will be translated to human fetuses within a few years. It’s imperative to deepen the study of these cells and the applicability of TRASCET, both in pre-clinical and clinical studies.
Description
Keywords
Anomalias Congénitas Células Estaminais Células Estaminais do Líquido Amniótico Líquido Amniótico Terapia com Células Estaminais do Líquido Amniótico Trascet