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. This book provides an overview of the current advances in the field of gene therapy and the methods that are being successfully applied in the manufacture of gene therapeutic products, and hopefully will stimulate further progress and advancement in this field to meet the ever-increasing demands.

lgrsnf/A:\compressed\10.1007%2F978-1-4615-1353-7.pdf

nexusstc/Manufacturing of Gene Therapeutics: Methods, Processing, Regulation, and Validation/44ceef22fa3d20e7275dfb9242980907.pdf

scihub/10.1007/978-1-4615-1353-7.pdf

zlib/Engineering/Mark Lawler (auth.), G. Subramanian (eds.)/Manufacturing of Gene Therapeutics: Methods, Processing, Regulation, and Validation_2094272.pdf

edited by G. Subramanian

First edition, New York, [New York, 2002

United States, United States of America

Springer Nature, New York, NY, 2012

Boston, MA, United States, 2002

2002, PS, 2012

lg940392

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Online full text is restricted to subscribers.
Also available in print.
Mode of access: World Wide Web.

Front Matter....Pages i-xvii
Somatic Gene Therapy, Paradigm Shift or Pandora’s Box....Pages 1-16
Gene Therapy for Cancer....Pages 17-32
Gene Self-Assembly (GENSA)....Pages 33-43
Gene Expression....Pages 45-57
Tumour Genotype and Response to Cytotoxic Gene Therapy....Pages 59-98
Protein Binding Matrices....Pages 99-133
Gene Transfer into Eukaryotic Cells....Pages 135-153
Plasmid DNA Manufacturing....Pages 155-168
Quality Assurance and Quality Control for Viral Therapeutics....Pages 169-200
Analytical Assays to Characterise Adenoviral Vectors and Their Applications....Pages 201-225
Validation of Gene Therapy Manufacturing Processes....Pages 227-244
Gene Delivery....Pages 245-272
Regulatory Issues in Gene Therapy....Pages 273-287
Regulatory Aspects in Gene Therapy....Pages 289-310
Regulatory Issues for Process Development and Manufacture of Plasmids Under Contract....Pages 311-330
Risk Assessment in Gene Therapy....Pages 331-338
Back Matter....Pages 339-345

2013-08-01