| Revista: | Genetics and molecular biology |
| Base de datos: | PERIÓDICA |
| Número de sistema: | 000459278 |
| ISSN: | 1415-4757 |
| Autores: | Freitas, Martiela Vaz de1 Frâncio, Lariane1 Haleva, Laura2 Matte, Ursula da Silveira1 |
| Instituciones: | 1Hospital de Clinicas de Porto Alegre, Centro de Pesquisa Experimental, Porto Alegre, Rio Grande do Sul. Brasil 2Universidade Federal do Rio Grande do Sul, Instituto de Biociencias, Porto Alegre, Rio Grande do Sul. Brasil |
| Año: | 2022 |
| Volumen: | 45 |
| Número: | 3 |
| País: | Brasil |
| Idioma: | Inglés |
| Tipo de documento: | Artículo |
| Enfoque: | Descriptivo |
| Resumen en inglés | There are many clinical trials underway for the development of gene therapies, and some have resulted in gene therapy products being commercially approved already. Significant progress was made to develop safer and more effective strategies to deliver and regulate genetic products. An unsolved aspect is the immune system, which can affect the efficiency of gene therapy in different ways. Here we present an overview of approved gene therapy products and the immune response elicited by gene delivery systems. These include responses against the vector or its content after delivery and against the product of the corrected gene. Strategies to overcome the hurdles include hiding the vector or/and the transgene product from the immune system and hiding the immune system from the vector/transgene product. Combining different strategies, such as patient screening and intelligent vector design, gene therapy is set to make a difference in the life of patients with severe genetic diseases |
| Disciplinas: | Biología, Medicina |
| Palabras clave: | Inmunología, Genética, Terapia génica, Revisión bibliográfica |
| Keyword: | Immunology, Genetics, Gene therapy, Bibliographic review |
| Texto completo: | Texto completo (Ver HTML) Texto completo (Ver PDF) |
