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Avanços recentes em terapia génica
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A terapia génica é um campo de estudo vasto e complexo que tem evoluído rapidamente nos últimos anos. Novas técnicas surgiram e outras foram aprimoradas, possibilitando a realização de ensaios clínicos que resultaram na aprovação de terapias génicas por respeitadas agências reguladoras, nomeadamente FDA e EMA. Desta forma, é indispensável esclarecer a comunidade médica sobre as técnicas em que assentam os progressos desta área de estudo. Neste trabalho, procura-se inicialmente clarificar os principais conceitos teóricos sobre as técnicas mais recentes de terapia génica, com enfase na utilização de vectores virais e sistemas CRISPR/Cas9. Considerando a terapia génica como a Medicina do futuro, o principal objectivo deste trabalho é perceber quais são as técnicas de edição genética mais utilizadas em ensaios clínicos actualmente em curso, bem como reconhecer os avanços alcançados, baseando a discussão numa revisão sistemática. Após a análise dos dados obtidos, pretende-se discutir de forma crítica sobre os obstáculos a transpor de modo a alcançar a manipulação assertiva e eficaz do genoma humano, bem como as implicações de descobertas recentes para a Medicina actual. O que trarão as terapias génicas ao futuro da Medicina?
Gene therapy is a rapidly evolving, vast, and complex field of study. In recent years, new techniques emerged and others were improved, allowing the design of gene therapy based clinical trials, that led already to the approval of some treatments for rare diseases by respected regulatory agencies, such as FDA and EMA. Clarifying the medical community about the techniques on which this progress was built is indispensable. In this work, I start by describing the theorical concepts behind gene therapy techniques, with an emphasis on viral vectors and CRISPR/Cas systems. Considering gene therapy as the future of Medicine, the main goal of this review is to understand which editing techniques are mostly used on clinical trials nowadays, as well as to recognize their results. A search was conducted to gather the most recent clinical trials using gene therapies. The analysis of results is expected to lead to a discussion about the barriers left to overcome to achieve an effective manipulation of the human genome, as well as the implications of recent discoveries to current medical practice. How is gene therapy shaping the future of Medicine?
Gene therapy is a rapidly evolving, vast, and complex field of study. In recent years, new techniques emerged and others were improved, allowing the design of gene therapy based clinical trials, that led already to the approval of some treatments for rare diseases by respected regulatory agencies, such as FDA and EMA. Clarifying the medical community about the techniques on which this progress was built is indispensable. In this work, I start by describing the theorical concepts behind gene therapy techniques, with an emphasis on viral vectors and CRISPR/Cas systems. Considering gene therapy as the future of Medicine, the main goal of this review is to understand which editing techniques are mostly used on clinical trials nowadays, as well as to recognize their results. A search was conducted to gather the most recent clinical trials using gene therapies. The analysis of results is expected to lead to a discussion about the barriers left to overcome to achieve an effective manipulation of the human genome, as well as the implications of recent discoveries to current medical practice. How is gene therapy shaping the future of Medicine?
Descrição
Trabalho Final do Curso de Mestrado Integrado em Medicina, Faculdade de Medicina, Universidade de Lisboa, 2021
Palavras-chave
Terapia génica Vectores virais CRISPR/Cas AAVs Ensaios clínicos