Medicina: Ematologia: Talassemie e trapianto del midollo osseo
| English |
| gene therapy |
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| Attestation |
3
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| Part of speech |
Noun syntagm
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| Grammatical label |
Countable
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| Definition |
Recent advances in molecular biology have resulted in the cloning of the normal genes involved in a number of human genetic diseases. To extend the benefits derived from BMT to patients without a histocompatible donor, studies have begun to evaluate gene therapy. Gene therapy is a proposed technique for the correction of a patient´s cells by the addition of a normal copy of the defective gene.
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| Definition source |
Touraine, JL., et al. (1999). ‘Stem cell transplants in utero for genetic diseases: treatment and a model for induction of immunologic tolerance’. Transplantation Proceedings 31(1-2):681-2 (RISCEN108)
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| Context |
Although most current experimental studies of gene therapy have focused on the use of bone marrow cells, alternative targets for gene therapy could include keratinocytes, hepatocytes, or fibroblasts. For gene therapy to have a sustained effect, the genetic correction must occur at the level of self-renewing, pluripotent lymphoid or hematopoietic stem cells.
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| Context source |
Touraine, JL., et al. (1999). ‘Stem cell transplants in utero for genetic diseases: treatment and a model for induction of immunologic tolerance’. Transplantation Proceedings 31(1-2):681-2 (RISCEN108)
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| Subject field |
Haemopoiesis
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| Sub-field (level 1) |
Thalassemias
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| Sub-field (level 2) |
Therapies
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| Coordinate concept |
Transfusional therapy, allogeneic bone marrow transplantation, syngeneic bone marrow transplantation, hematopoietic stem cell transplantation, iron-chelation therapy
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| Related concept |
Globin gene, viral vector, retrovirus, lentivirus, adeno-associated virus, herpes simplex, gene expression, gene transfection
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| it |
Terapia genica
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| Reliability code |
3
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