Year: 1999
Type: dissertation
Abstract: Gene therapy is a procedure in which exogenous genetic material is\nintroduced into the cells of a patient in order to correct an genetic error or\nto provide the cells of the patient with a new functional property.\nCorrection can be achieved by gene targeting via homologous\nrecombination, at present achieved with very low efficiency (reviewed in\nYanez and Porter 1998), or by addition of a therapeutic gene\n(augmentation). For gene therapy to succeed in clinical practice, the\ntherapeutic gene must be delivered efficiently into the appropriate cells\n(tissue) and, once delivered, the gene must be expressed at a therapeutical\nlevel.\nIn this chapter (section A) I will summarise some of the efforts currently\nunderway for the development of practical, efficient and safe methods for\ngene transfer in man and the rationale underlying these approaches as well\nas the limitations and the problems involved (Tables IA and IB). I will\nalso address the following issues: regulation of gene expression (section\nB), possible targets for gene therapy (section C), creation of animal models\nfor human diseases and their relevance to somatic gene therapy (section D)\nand ethical and social implications of gene therapy (section E).
Author: Dubravka Drabek
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: green
OpenAlex ID: W1744852346