Gene-targeting applications

Table 2.1. Sequence context of mutation types and their frequencies observed after application of error-prone PCR as described herein and sequencing of cloned genes. Targets T7 RNAP, coding sequence of T7 RNA polymerase, Poll, coding sequence of E. coli DNA polymerase I, Inti on, cDNA of Tetrahymena thermophila intron,...

PEI grafting on chitosan is becoming a popular approach for modification of chitosan for gene delivery applications. In recent years several efforts to develop chitosan-g-PEI delivery vectors have been published. These include incorporation of mannose [117] and folic acid [118] derivatives for targeted delivery and application of chitosan-g-PEI for the delivery of siRNA [119]. 

Most gene therapy applications require extravasation of the DNA carriers so that only relative small DNA complexes can pass through the blood vessels and interact directly with parenchymal cells after vascular administration [2]. Under pathophysiological conditions, the structure of the vasculature can change. This phenomenon - termed the enhanced permeation effect - has been utilized to passively target macromolecules to tumors, since blood vessels in tumors are relatively more leaky. 

Gu, H. (1994) Gene targeting and its application to the study of B-cell development. Curr. Opin. Immunol. 6, 308-312. 

DBD Regulatory domain Gene target Experimental model Therapeutic application References... 

Urban-Klein B, Werth S, Abuharbeid S, Czubayko F, Aigner A (2005) RNAi-mediated gene-targeting through systemic application of polyethylenimine (PEI)-complexed siRNA in vivo. Gene Ther 12(5) 461 66... 

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