Gene Therapy of the Central Nervous System: From Bench to by Michael G. Kaplitt, Matthew During

By Michael G. Kaplitt, Matthew During

Few components of biomedical study offer better possibilities for significantly new cures for devastating illnesses that experience kept away from remedy to this point than gene remedy. this is often fairly real for the mind and worried process, the place gene move has turn into a key know-how for simple study and has lately been translated to human remedy in different landmark medical trials. Gene remedy of the vital anxious process: From Bench to Bedside represents the 1st definitive quantity in this topic. Edited through pioneers of neurological gene treatment, this quantity comprises contributions via leaders who helped create this box and are increasing the promise of gene treatment for the way forward for easy and scientific neuroscience. Drawing upon this wide collective event, this e-book presents transparent and informative experiences on a number of matters of curiosity to someone exploring or utilizing gene remedy for neurobiological functions in study and scientific praxis. * provides gene move applied sciences with specific emphases upon novel cars, immunological matters and the position of gene remedy in stem cells * Discusses preclinical parts which are more likely to translate into scientific reports within the close to destiny, together with epilepsy, soreness and amyotrophic lateral sclerosis * contains ''insider'' info on technological and regulatory concerns which may frequently restrict potent translation of even the main promising suggestion into medical use

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

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1999). This intercellular movement can be visualized with a fluorescent protein-tagged tegument protein (Elliott and O'Hare, 1999a, b). , 2000). , submitted). Capsid proteins can also be used to label virions and target proteins to the nucleus. The capsid protein VP26 was fused to GFP and incorporated into virions (Desai and Person, 1998). GFP did not affect the cellular distribution of VP26 and was detected within the nucleus within minutes after infection. These experiments indicate that therapeutic and imaging proteins fused to capsid or tegument proteins have the potential to be transported efficiently to the nucleus and to carry out functional activities.

These include: (i) the development of packaging systems that allow the preparation of vector stocks with little or no helper virus contamination; (ii) the inclusion of elements from other viruses that confer physical stability to HSV amplicon vector o virus replication (cell death and vector production) (stable episome) PERSPECTIVES transduction (death due to therapeutic gene) (not toxic to neurons) FIGURE 5 HSV vectors for brain tumor therapy. For tumor therapy vectors are designed to be selectively toxic to dividing cells, as most normal cells in the brain are not dividing.

Methods, 33:179-186. , Manservigi, R. O. (2004a) HSV-1 virions engineered for specific binding to cell surface receptor. , 9: 419-427. , Baumeister, W and Steven, A. C. (2003) Three-dimensional structure of herpes simplex virus from cryo-electron tomography. Science, 302: 1396-1398. Hammerschmidt, W. and Sugden, B. (2004) Epstein-Barr virus sustains Burkitt's lymphomas and Hodgkin's disease. Trends Mol. , 10: 331-336. , Sena-Esteves, M. O. (2003) Potentiated gene delivery to tumor using herpes simplex virus/Epstein Barr/RV tribrid amplicon vectors.

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