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File Format | PDF
File Size | 16.8 MB
Pages | 366
Language | English
Category | Pharmacology
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Description: Advances in
molecular biology and recombinant DNA technology have accelerated progress in
many fields of life science research, including gene therapy. A large number of
genetic engineering approaches and methods are readily available for gene
cloning and therapeutic vector construction. Significant progress is being made
in genomic, DNA sequencing, gene expression, gene delivery and cloning. Thus
gene therapy has already shown that it holds great promise for the treatment of
many diseases and disorders. In general it involves the delivery of recombinant
genes or transgenes into somatic cells to replace proteins with a genetic
defect or to transfer with the pathological process of an illness. The viral
and non-viral delivery systems may hold the potential for future non-invasive,
cost-effective oral therapy of genetically-based disorders.
Recent years
have seen considerable progress in the discovery and early clinical development
of a variety of gene therapeutic products. The availability, validation, and
implementation of gene therapeutic products has also enabled success in testing
and evaluation. New challenges will need to be overcome to ensure that products
will also be successful in later clinical development and ultimately for
marketing authorisation. These new challenges will include improvements in
delivery systems, better control of in-vivo targeting, increased level
transduction and duration of expression of the gene, and manufacturing process
efficiencies that enable reduction in production costs. Perhaps profound
understanding of regulated gene design may result in innovative bioproducts
exhibiting safety and efficacy profiles that are significantly superior to
those achieved by the use of naturally occurring genes. This procedure may
contribute considerably to fulfilling standards set by regulatory authorities.
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Manufacturing of Gene Therapeutics: Methods, Processing, Regulation and Validation
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