Gene Therapy – Technologies, Markets and Companies 2013

Posted: June 17, 2013 at 1:47 pm

DUBLIN, June 17, 2013 /PRNewswire/ --

Research and Markets (http://www.researchandmarkets.com/research/dfcq6z/gene_therapy) has announced a new report "Gene Therapy - Technologies, Markets and Companies" to its offering.

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Gene therapy can be broadly defined as the transfer of defined genetic material to specific target cells of a patient for the ultimate purpose of preventing or altering a particular disease state. Genes and DNA are now being introduced without the use of vectors and various techniques are being used to modify the function of genes in vivo without gene transfer. If one adds to this the cell therapy particularly with use of genetically modified cells, the scope of gene therapy becomes much broader. Gene therapy can now be combined with antisense techniques such as RNA interference (RNAi), further increasing the therapeutic applications. This report takes a broad overview of gene therapy and is the most up-to-date presentation from the author on this topic built-up from a series of gene therapy reports written by him during the past decade, including a textbook of gene therapy and a book on gene therapy companies. This report describes the setbacks of gene therapy and renewed interest in the topic.

Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration, as well as targeted gene therapy, are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.

Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included.

Research and development is in progress in both the academic and the industrial sectors. The National Institutes of Health (NIH) of the US is playing an important part. As of 2012, over 2030 clinical trials have been completed, are ongoing or have been approved worldwide. A breakdown of these trials is shown according to the areas of application.

The voluminous literature on gene therapy was reviewed and selected 750 references are appended in the bibliography. The references are constantly updated. The text is supplemented with 73 tables and 15 figures.

Profiles of 181 companies involved in developing gene therapy are presented, along with 204 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade. These companies have been followed up since they were the topic of a book on gene therapy companies by the author of this report. John Wiley & Sons published the book in 2000 and from 2001 to 2003, updated versions of these companies (approximately 160 at mid-2003) were available on Wiley's web site. Since that free service was discontinued and the rights reverted to the author, this report remains the only authorized continuously updated version on gene therapy companies.

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Gene Therapy - Technologies, Markets and Companies 2013

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