Read e-book online Gene Therapy of the Central Nervous System: From Bench to PDF

By Michael G. Kaplitt, Matthew During

ISBN-10: 0080454372

ISBN-13: 9780080454375

ISBN-10: 0123976324

ISBN-13: 9780123976321

Few parts of biomedical examine offer larger possibilities for noticeably new cures for devastating ailments that experience kept away from therapy to this point than gene treatment. this is often fairly actual for the mind and apprehensive approach, the place gene move has develop into a key know-how for simple study and has lately been translated to human remedy in numerous landmark scientific trials. Gene treatment of the primary anxious approach: From Bench to Bedside represents the 1st definitive quantity in this topic. Edited by means of pioneers of neurological gene treatment, this quantity includes contributions by way of leaders who helped create this box and are increasing the promise of gene remedy for the way forward for easy and scientific neuroscience. Drawing upon this wide collective event, this publication presents transparent and informative experiences on various topics of curiosity to somebody exploring or utilizing gene remedy for neurobiological functions in learn and scientific praxis. * provides gene move applied sciences with specific emphases upon novel automobiles, immunological matters and the position of gene treatment in stem cells * Discusses preclinical parts which are more likely to translate into scientific reviews within the close to destiny, together with epilepsy, soreness and amyotrophic lateral sclerosis * comprises ''insider'' info on technological and regulatory matters that may usually restrict potent translation of even the main promising concept into medical use

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Extra resources for Gene Therapy of the Central Nervous System: From Bench to Bedside

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2002). HSV/AAV amplicon vectors hold great promise for stable delivery of these large cDNAs or genomic fragments into target regions of the genome for sustained restoration of function. , 2005). In initial studies, peripheral administration of A-j8 expressing vectors was found to decrease A-P deposition in the brains of transgenic mice overexpressing this protein, albeit with some associated inflammatory response. HSV amplicon vectors have also been explored as a means of treatment for experimental brain tumors.

1997) HSV/AAV hybrid amplicon vectors extend transgene expression in human glioma cells. Hum. , 8: 359-370. E. and Kaptein, R. (2005) Structural properties of the promiscuous VP16 activation domain. Biochemistry, 44: 827-839. Kaneda, Y, Twai, K. and Uchida, T. (1989) Increased expression of DNA cointroduced with nuclear protein in adult rat liver. Science, 243: 375-378. D. D.

A. and Basilion, J. R (2002) MRI of transgene expression: correlation of therapeutic gene expression. Neoplasia, 4: 523-530. A. and Wade-Martins, R. (2004) Infectious delivery of the 132 kb CDKN2A/CDKN2B genomic DNA region results in correctly spliced gene expression and growth suppression in glioma cells. , 11: 1195-1204. M. G. (1990) Molecular and biological characterization of a herpes simplex virus type 1 (HSV-1) neuroinvasiveness gene. J. Exp. , 172: 487-496. , Wienhard, K. D. (2003) Improved herpes simplex virus type 1 amplicon vectors for proportional coexpression of positron emission tomography marker and therapeutic genes.

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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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