Concepts in Genetic Medicine by Boro Dropulic, Barrie Carter

By Boro Dropulic, Barrie Carter

This publication grants a set of prepared and succinct studies within the box of healing genetic drugs offered via a gently chosen crew of most sensible specialists. every one bankruptcy specializes in a unmarried, present subject and explains matters within the improvement of genetic remedies, serious demanding situations and techniques for imposing them, and destiny instructions in study and translational functions. The booklet is complemented with uniformly designed illustrations and a supplementary site with a PowerPoint presentation for educators.

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30 SIV-derived vectors are inefficient in human cells, as are HIV-derived vectors in simian cells, and therefore 22 LENTIVIRUS VECTORS SIV vectors have been derived primarily for development of nonhuman primate modeling for lentiviral vector applications. S. Food and Drug Administration (FDA) in 2001 prior to initiation of the first clinical trial evaluating a lentiviral vector. 8 Use of an HIV-derived vector first in HIV-infected persons helped to move clinical application forward since the risk/benefit ratio in these patients is more favorable.

2004;6:S67–S82. 33. Delenda C. Lentiviral vectors: optimization of packaging, transduction and gene expression. J Gene Med. 2004;6:S125–S138. 34. Baum C, Schambach A, Bohne J, Galla M. Retrovirus vectors: toward the plentivirus? Mol Ther. 2006;13(6):1050–1063. 35. Sinn PL, Sauter SL, McCray PL. Gene therapy progress and prospects: development of improved lentiviral and retroviral vectors—design, biosafety, and production. Gene Ther. 2005;12:1089–1098. 36. Couture LA, Mullen CA, Morgan RA. Retroviral vectors containing chimeric promoter/enhancer elements exhibit cell-type-specific gene-expression.

2003; 02-0824fje. 103. Tschernutter M, Schlichtenbrede FC, Howe S, et al. Long-term preservation of retinal function in the RCS rat model of retinitis pigmentosa following lentivirus-mediated gene therapy. Gene Ther. 2005;12:694–701. 104. Kang Y, Xie L, Tran DT, et al. Persistent expression of factor VIII in vivo following nonprimate lentiviral gene transfer. Blood . 2005;106:1552–1558. 36 LENTIVIRUS VECTORS 105. Hanawa H, Hargrove PW, Kepes S, et al. Extended β-globin locus control region elements promote consistent therapeutic expression of a γ-globin lentiviral vector in murine β-thalassemia.

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