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- W1744852346 abstract "textabstractGene therapy is a procedure in which exogenous genetic material isintroduced into the cells of a patient in order to correct an genetic error orto provide the cells of the patient with a new functional property.Correction can be achieved by gene targeting via homologousrecombination, at present achieved with very low efficiency (reviewed inYanez and Porter 1998), or by addition of a therapeutic gene(augmentation). For gene therapy to succeed in clinical practice, thetherapeutic gene must be delivered efficiently into the appropriate cells(tissue) and, once delivered, the gene must be expressed at a therapeuticallevel.In this chapter (section A) I will summarise some of the efforts currentlyunderway for the development of practical, efficient and safe methods forgene transfer in man and the rationale underlying these approaches as wellas the limitations and the problems involved (Tables IA and IB). I willalso address the following issues: regulation of gene expression (sectionB), possible targets for gene therapy (section C), creation of animal modelsfor human diseases and their relevance to somatic gene therapy (section D)and ethical and social implications of gene therapy (section E)." @default.
- W1744852346 created "2016-06-24" @default.
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- W1744852346 date "1999-09-29" @default.
- W1744852346 modified "2023-09-24" @default.
- W1744852346 title "Locus control regions and gene therapy" @default.
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