By Boro Dropulic, Barrie Carter
This booklet provides a set of prepared and succinct stories within the box of healing genetic medication provided by way of a gently chosen staff of best specialists. every one bankruptcy makes a speciality of a unmarried, present subject and explains matters within the improvement of genetic remedies, severe demanding situations and methods for enforcing them, and destiny instructions in study and translational purposes. The publication is complemented with uniformly designed illustrations and a supplementary website with a PowerPoint presentation for educators.
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Extra info for Concepts in Genetic Medicine
Inducible expression systems are also useful for protein production in biotechnology. 122,123 Protein manufacture can therefore be enhanced if protein expression can be limited to the period of production. The efﬁciency, stability, and long-term gene expression of lentiviral vectors makes them a very useful tool for this area of biotechnology. For example, difﬁcult to manufacture proteins such as factor VIII could be produced more cost-effectively by using lentiviral vectors both to decrease the time to generate high-expressing cell clones and to increase protein yield.
The goals for vector modiﬁcation are typically to increase titers, heighten biosafety, improve transduction efﬁciency, and promote high and durable gene expression. These goals have been addressed primarily through the inclusion of cis-acting sequences, degeneration and minimization of vector sequences whenever possible, pseudotyping with appropriate envelopes, and application of efﬁcient promoters. As the ﬁeld progresses, vectors have evolved to exhibit new characteristics, such as efﬁcient dual-gene expression, tissue or cell speciﬁcity, episomal gene expression (nonintegrating vectors), or RNA-only expression.
17. Mitrophanous KA, Yoon S, Rohll JB, et al. Stable gene transfer to the nervous system using a non-primate lentiviral vector. Gene Ther. 1999;6:1808–1818. 18. Molina RP, Matukonis M, Paszkiet B, et al. Mapping of the bovine immunodeﬁciency virus packaging signal and RRE and incorporation into a minimal gene transfer vector. Virology. 2002;304:10–23. 19. Matukonis M, Li M, Molina RP, et al. Development of second- and third-generation bovine immunodeﬁciency virus-based gene transfer systems. Hum Gene Ther.