| Journal: | Brazilian journal of medical and biological research |
| Database: | PERIÓDICA |
| System number: | 000350310 |
| ISSN: | 0100-879X |
| Authors: | Strauss, B.E1 Costanzi-Strauss, E2 |
| Institutions: | 1Universidade de Sao Paulo, Faculdade de Medicina, Sao Paulo. Brasil 2Universidade de Sao Paulo, Instituto de Ciencias Biomedicas, Sao Paulo. Brasil |
| Year: | 2007 |
| Season: | May |
| Volumen: | 40 |
| Number: | 5 |
| Pages: | 601-613 |
| Country: | Brasil |
| Language: | Inglés |
| Document type: | Artículo |
| Approach: | Caso clínico |
| English abstract | A successful gene therapy clinical trial that also encountered serious adverse effects has sparked extensive study and debate about the future directions for retrovirus-mediated interventions. Treatment of X-linked severe combined immunodeficiency with an oncoretrovirus harboring a normal copy of the gc gene was applied in two clinical trials, essentially curing 13 of 16 infants, restoring a normal immune system without the need for additional immune-related therapies. Approximately 3 years after their gene therapy, tragically, 3 of these children, all from the same trial, developed leukemia as a result of this experimental treatment. The current understanding of the mechanism behind this leukemogenesis involves three critical and cooperating factors, i.e., viral integration, oncogene activation, and the function of the therapeutic gene. In this review, we will explore the causes of this unwanted event and some of the possibilities for reducing the risk of its reoccurrence |
| Disciplines: | Medicina |
| Keyword: | Oncología, Genética, Retrovirus, Mutagénesis, Síndrome de inmunodeficiencia combinada severa, Terapia génica |
| Keyword: | Medicine, Oncology, Retrovirus, Mutagenesis, Severe combined immunodeficiency syndrome, Gene therapy, Genetics |
| Full text: | Texto completo (Ver HTML) |
