This text presents step-by-step protocols for use in gene therapy, describing methods for working with specific delivery systems and viral vectors used extensively in this field. Each chapter in this provides a basic description of how best to utilize each vector system, and espouses on the advantages and disadvantages of the different vector systems.

Product Description: Leading gene therapy researchers and clinicians illuminate the field-from basic vector technology to current and future clinical applications in neurology. The authoritative contributors provide cutting-edge reviews of the vectors available for gene transfer to the central nervous system, the strategies against CNS tumors, the potential strategies against neurologic disorder, and the limitations of today's gene therapy approaches...read more

Gene Therapy Technologies, Applications and Regulations From Laboratory to Clinic Edited by Anthony Meager Division of Immunobiology, The National Institute for Biological Standards and Control, South Mimms, UK The development of gene-based technologies has been rapid over the past decade and has consequently resulted in a surge of interest in human gene therapy, the deliberate transfer of genes to somatic cells to cure or alleviate disease symptoms. Hundreds of clinical protocols involving variously designed vectors for efficient gene transfer have been developed. However, the use of such complex 'gene medicines' containing potentially heritable genes has raised numerous concerns regarding quality, efficacy and safety. Encompassing recent developments in the field and addressing current concerns this book: * surveys many of the current technologies for preparing vectors for use in gene therapy protocols * reviews the application of gene-mediated therapies to a range of medical conditions * considers the regulatory aspects of gene therapy including product quality and safety requirements * appraises the transfer of technologies from laboratory to clinic with regard to the attendant requirements and facilities for: * good laboratory practice (GLP) conditions in the R&D laboratory * large-scale production methods and good manufacturing practice (GMP) * current in-process and final product testing Written by international experts knowledgeable about many aspects of human somatic gene therapy, this book will be an essential guide for those embarking on gene therapy technologies relevant to specifications of production and testing of products (and procedures) required to meet existing regulations, including quality, efficacy and safety considerations.

Product Description: Genes, which are carried on chromosomes, are the basic physical and functional units of heredity. Genes are specific sequences of bases that encode instructions on how to make proteins. Although genes get a lot of attention, it's the proteins that perform most life functions and even make up the majority of cellular structures...read more

Product Description: Several years ago, when the discovery of catalytic RNA was recognized in a public manner,many people asked if new ?elds of therapy would soon be available. Although some tentative positive answers were given,nobody would say with certainty that RNA of various kinds was a truly promising means of altering gene expression...read more

Product Description: Coedited by internationally recognized leaders in gene therapy research, this guide supplies the most recent advances, studies, and expert opinion on gene therapy for neurological disorders. Spanning conditions such as Parkinsons's and Alzheimer's disease, multiple sclerosis, brain tumors, stroke, epilepsy, and brain and spinal cord trauma, this reference presents the basic science and applications of recombinant gene products, immune and stem cell-mediated therapeutic strategies, and brain tumor gene therapy...read more

Product Description: Despite various difficulties, the field of gene therapy, particularly with regard to cancer, has accumulated a tremendous amount of vital pre-clinical and clinical data. "Gene Therapy of Cancer: Methods and Protocols, Second Edition" fully updates the first edition with expert coverage of established and novel protocols involving both experimental and clinical approaches to cancer gene therapy...read more

Gene therapy has expanded rapidly over the last decade. The number of clinical trials reported by 2001 included 532 protocols and 3436 patients. Phase I trials predominate with 359 trials of 1774 patients versus Phase II (57 trials with 507 patients) and Phase III (3 trials of 251 patients). The disease overwhelmingly targeted by gene therapy is cancer: involving 331 trials with 2361 patients. Despite the somewhat disappointing results of clinical trials to date, gene therapy offers tremendous promise for the future of cancer therapy. The area of gene therapy is vast, and both malignant and nonmalignant cells can be targeted. Suicide Gene Therapy: Methods and Reviews covers gene therapy that targets malignant cells in a treatment that has become known as “suicide gene therapy. ” Basically, this approach uses the transduction of cancer cells with a gene for a foreign enzyme that, when expressed, is able to activate a nontoxic prodrug into a highly cytotoxic drug able to kill the cancer cell population. This is a major area in cancer gene therapy―in 2001 this technique was represented by 52 clinical protocols with a total of 567 patients. Additional trials used multiple gene therapy protocols that also involved suicide gene therapy (83 with 497 patients), indicating that the interest in this area is considerable. Suicide Gene Therapy: Methods and Reviews aims to cover comprehensively, both in theoretical and practical terms, the rapidly evolving area of suicide gene therapy for cancer.

During the first half century of genetics, coinciding with the first half of this cen­ tury, geneticists dreamt of the repair of genetic disease by altering or replacing defective genes. H. J. Muller wrote of the great advantages of mutations, "nanoneedles" in his apt term, for delicately probing physiological and chemical processes. In the same spirit, genes could be used to provide treatments of needle point delicacy. Yet, during this period no realistic possibility appeared; it remained but a dream. The situation changed abruptly at the half century. Microbial genetics and its offshoot, cell culture genetics, provided the route. Pneumococcus transformation showed that exogenous DNA could become a permanent part of the genome; yet attempts to reproduce this in animals produced a few tantalizing hints of success, but mostly failures. Transduction, using a virus as mediator, offered a better op­ portunity. The fITSt reproducible in vivo gene therapy in a whole animal came in 1981. This was in Drosophila, with a transposable element as carrier. Flies were "cured" of a mutant eye color by incorporation of the normal allele, and the effect was transmissible, foreshadowing not only somatic, but germ line gene therapy. At the same time, retroviruses carrying human genes were found to be ex­ tremely efficient in transferring their contents to the chromosomes of cultured cells.