By Michael G. Kaplitt, Matthew During
ISBN-10: 0080454372
ISBN-13: 9780080454375
ISBN-10: 0123976324
ISBN-13: 9780123976321
Few components of biomedical study offer larger possibilities for extensively new remedies for devastating ailments that experience kept away from therapy to this point than gene treatment. this is often relatively precise for the mind and worried procedure, the place gene move has turn into a key know-how for simple examine and has lately been translated to human treatment in numerous landmark medical trials. Gene treatment of the vital worried approach: From Bench to Bedside represents the 1st definitive quantity in this topic. Edited by way of pioneers of neurological gene remedy, this quantity comprises contributions by means of leaders who helped create this box and are increasing the promise of gene treatment for the way forward for uncomplicated and scientific neuroscience. Drawing upon this large collective event, this e-book offers transparent and informative stories on a number of matters of curiosity to someone exploring or utilizing gene remedy for neurobiological functions in examine and scientific praxis. * offers gene move applied sciences with specific emphases upon novel autos, immunological concerns and the function of gene treatment in stem cells * Discusses preclinical components which are more likely to translate into scientific experiences within the close to destiny, together with epilepsy, discomfort and amyotrophic lateral sclerosis * contains ''insider'' details on technological and regulatory matters which may usually restrict potent translation of even the main promising thought into medical use
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4). g. , 2002). Cell type-specific, and frequently longer term, expression can be achieved using mammalian promoters specific to the cell of interest. , 1999), and a hybrid promoter combining elements of the ENK promoter with that of the neurofilament heavy chain in striatal neurons (Wang et a l , 2004). , 2003). , 2002). , 2000). , 2002). , 2005). The development of informative imaging reporters now allows refined assessment of intracellular dynamics, as well as parameters of gene delivery in vivo.
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Gene Therapy of the Central Nervous System: From Bench to Bedside by Michael G. Kaplitt, Matthew During
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