Download Concepts in Genetic Medicine by Boro Dropulic, Barrie Carter PDF

By Boro Dropulic, Barrie Carter

This ebook provides a set of geared up and succinct studies within the box of healing genetic drugs provided by means of a delicately chosen crew of most sensible specialists. each one bankruptcy specializes in 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 learn and translational functions. The ebook is complemented with uniformly designed illustrations and a supplementary site with a PowerPoint presentation for educators.

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REFERENCES 1. Miller AD, Eckner RJ, Jolly DJ, Friedmann T, Verma IM. Expression of a retrovirus encoding human HPRT in mice. Science. 1984;230:1395–1398. 2. Williams DA, Orkin SH, Mulligan RC. Retrovirus-mediated transfer of human adenosine deaminase gene sequences into cells in culture and into murine hematopoietic cells in vivo. Proc Natl Acad Sci U S A. 1986;83:2566–2570. 3. Eglitis MA, Kantoff P, Gilboa E, Anderson WF. Gene expression in mice after high efficiency retroviral-mediated gene transfer.

Brun S, Faucon-Biguet N, Mallet J. Optimization of transgene expression at the posttranscriptional level in neural cells: implications for gene therapy. Mol Ther. 2003;7:782–789. 74. Pfeifer A, Ikawa M, Dayn Y, Verma IM. Transgenesis by lentiviral vectors: lack of gene silencing in mammalian embryonic stem cells and preimplantation embryos. Proc Natl Acad Sci U S A. 2002;99:2140–2145. 75. Lois C, Hong EJ, Pease S, Brown EJ, Baltimore D. Germline transmission and tissue-specific expression of transgenes delivered by lentiviral vectors.

60 Another alternative to the IRES is the use of self-cleaving 2A cleavage factors, which are derived from various picornaviruses. 62 Important cis-Acting Elements In addition to promoters, the packaging signal (ψ), integration signals (attL and attR), primer binding site and splice donor, and in most cases, splice acceptor sites (SD and SA) are required elements. 65 Most often, the rev response element (RRE) is used, which requires incorporation of Rev in the production system. RRE can be replaced with alternative RNA export elements, such as the constitutive transport element (CTE) derived from the beta retroviruses and simian retrovirus type 1 (SRV-1).

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