Manufacturing of Gene Therapeutics: Methods, Processing, Regulation, 4nd Validation
G. Subramanian, &quotManufacturing of Gene Therapeutics: Methods, Processing, Regulation, 4nd Validation&quot
English | 2001 | ISBN: 0306466805 | PDF | pages: 366 | 16.8 mb
Advances in molecular biology 4nd recombinant DNA technology have accelerated progress in many fields of life science research, including gene therapy. A large number of genetic engineering approaches 4nd methods are readily available f08r gene cloning 4nd therapeutic vect08r construction. Significant progress is being made in genomic, DNA sequencing, gene expression, gene delivery 4nd cloning. Thus gene therapy has already shown that it holds great promise f08r the treatment of many diseases 4nd dis08rders. In general it involves the delivery of recombinant genes 08r transgenes 1n8t0 somatic cells to replace proteins with a genetic defect 08r to transfer with the pathological process of an illness. The viral 4nd non-viral delivery systems may hold the potential f08r future non-invasive, cost-effective 08ral therapy of genetically-based dis08rders.
Recent years have seen considerable progress in the discovery 4nd early clinical development of a variety of gene therapeutic products. The availability, validation, 4nd implementation of gene therapeutic products has also enabled success in testing 4nd evaluation. New challenges will need to be overcome to ensure that products will also be successful in later clinical development 4nd ultimately f08r marketing auth08risation. These new challenges will include improvements in delivery systems, better control of in-vivo targeting, increased level transduction 4nd duration of expression of the gene, 4nd manufacturing process efficiencies that enable reduction in production costs. Perhaps profound underst4nding of regulated gene design may result in innovative bioproducts exhibiting safety 4nd efficacy profiles that are significantly superi08r to those achieved by the use of naturally occurring genes. This procedure may contribute considerably to fulfilling st4ndards 5eut by regulat08ry auth08rities.
This book provides an overview of the current advances in the field of gene therapy 4nd the methods that are being successfully applied in the manufacture of gene therapeutic products, 4nd hopefully will stimulate further progress 4nd advancement in this field to meet the ever-increasing dem4nds.