By Boro Dropulic, Barrie Carter
This e-book provides a set of prepared and succinct experiences within the box of healing genetic medication provided by way of a gently chosen team of best specialists. every one bankruptcy makes a speciality of a unmarried, present subject and explains concerns within the improvement of genetic remedies, serious demanding situations and methods for enforcing them, and destiny instructions in examine and translational functions. The publication is complemented with uniformly designed illustrations and a supplementary site with a PowerPoint presentation for educators.
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Additional info for Concepts in Genetic Medicine
Uk/genmed/ clinical). The safety database accrued from these trials is anticipated to help the general ﬁeld of clinical application of lentiviral vectors to move forward, and it is anticipated that non-HIV trials will begin soon in some of the areas described next. Since the ﬁrst report of lentiviral vectors working successfully in culture,1 there has been an explosion in use of the vectors for preclinical development of gene therapeutics. In general, these applications take advantage of the durability of the lentiviral vector expression to treat disorders that beneﬁt from long-term survival of cells modiﬁed for a lasting therapeutic effect.
BINDER Abramson Family Cancer Research Institute, School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania BORO DROPULIC Lentigen Corporation,, Baltimore, Maryland The ﬁeld of lentiviral vectors is a rapidly expanding and dynamic area of leadingedge science. 1 Within six years, in 2003, the ﬁrst lentiviral vector clinical trial had been opened for enrollment, and at the time this book went to press, nine trials have been opened. 1), to set the stage for more-in-depth chapters.
Zhu Y, Feuer G, Day SL, Wrzesinski S, Planelles V. Multigene lentiviral vectors based on differential splicing and translational control. Mol Ther. 2001;4:375–382. 59. Yu XB, Zhan XC, D’Costa J, et al. Lentiviral vectors with two independent internal promoters transfer high-level expression of multiple transgenes to human hematopoietic stem-progenitor cells. Mol Ther. 2003;7:827–838. 60. Amendola M, Venneri MA, Bifﬁ A, Vigna E, Naldini L. Coordinate dual-gene transgenesis by lentiviral vectors carrying synthetic bidirectional promoters.
Concepts in Genetic Medicine by Boro Dropulic, Barrie Carter