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Gene Therapy - technologies, markets and companies Published on September 2011
Report Summary 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 combined with antisense techniques such as RNA interference (RNAi), further increasing the therapeutic applications. This report takes 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 report 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 January 2010, over 2024 gene therapy 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. Since the death of Jesse Gelsinger in the US following a gene therapy treatment, the FDA has further tightened the regulatory control on gene therapy. A further setback was the reports of leukemia following use of retroviral vectors in successful gene therapy for adenosine deaminase deficiency. Several clinical trials were put on hold and many have resumed now. The report also discusses the adverse effects of various vectors, safety regulations and ethical aspects of gene therapy including germline gene therapy. The markets for gene therapy are difficult to estimate as there is only one approved gene therapy product and it is marketed in China since 2004. Gene therapy markets are estimated for the years 2010-2020. The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades. In spite of some setbacks, the future for gene therapy is bright.The markets for DNA vaccines are calculated separately as only genetically modified vaccines and those using viral vectors are included in the gene therapy markets The voluminous literature on gene therapy was reviewed and selected 700 references are appended in the bibliography.The references are constantly updated. The text is supplemented with 72 tables and 13 figures. Profiles of 188 companies involved in developing gene therapy are presented along with 207 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
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Table of Content TABLE OF CONTENTS 0. Executive Summary 19 1. Introduction 21 Definitions 21 Historical evolution of gene therapy 21 Relation of gene therapy to other biotechnologies 23 Molecular biological basics for gene therapy 23 Genome 23 DNA 24 RNA 24 Alternative RNA splicing 25 Genes 26 Gene regulation 26 Gene expression 28 Chromosomes 28 Telomeres 29 Mitochondrial DNA 29 Proteins 30 2. Gene Therapy Technologies 31 Classification of gene therapy techniques 31 Ex vivo and in vivo gene therapy 32 Ex vivo gene therapy 32 In vivo gene therapy 33 Physical methods of gene transfer 33 Electroporation 33 Applications of electroporation 34 Clinical applications of electroporation 35 Advantages of electroporation 35 Limitations of electroporation 36 Hydrodynamic 36 Microinjection 36 Particle bombardment 37 Ultrasound-mediated transfection 39 Molecular vibration 39 Application of pulsed magnetic field and superparamagnetic nanoparticles 39 Gene transfection using laser irradiation 40 Photochemical transfection 40 Chemical methods of gene transfer 41 Gene repair and replacement 41
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