Genetics gene therapy

GENESEQ GENESIS Gene therapy Genetic code Genetic colonization Genetic engineering... 

Gene therapy uses cloned DNA in adeno or retroviral vectors to transduce host cells for longterm therapy of certain genetic disorders with defined... 

DNA Vaccination and Genetic Vaccination Gene-therapy Vectors... 

Pfeifer A, Verma IM (2001) Gene therapy promises and problems. Annu Rev Genomics Hum Genet 2 177-211... 

The Competent Authorities are allowed a maximum of 60 days to review the data, or 90 days in the case of trials involving medicinal products for gene therapy, somatic cell therapy (including xenogeneic cell therapy), and all medicinal products containing genetically modified organisms. Such trials require written authorisation from the Competent Authority as distinct from other types of product where the authorities may just notify the sponsor of the acceptability (no-objection) of the application. If issues are raised, the sponsor may amend the application once, with a consequent extension to the allowed review period. 

Karpati, G., Ascadi, G. (1993). The potential for gene therapy in Ducheiuie muscular dystrophy and other genetic muscle diseases. Muscle Nerve 16, 1141-1153. 

An additional consideration that may influence the protocol used is the desired duration of subsequent expression of the gene product. In most cases of genetic disease, long-term expression of the inserted gene would be required. In other instances (e.g. some forms of cancer therapy or the use of gene therapy to deliver a DNA-based vaccine), short-term expression of the gene introduced would be sufficient/desirable. 

Although simple in concept, the application of gene therapy to treat/cure genetic diseases has, thus far, made little impact in practice. The slow progress in this regard is likely due to a number of factors. These include ... 

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