By A. Benigni, G. Remuzzi, C. Ronco
In the beginning conceived as a technique to treatment inherited genetic problems, gene treatment has been effectively utilized within the final decade to ameliorate the renal functionality compromised through revolutionary renal illnesses and to avoid kidney allograft rejection in experimental animals. within the current booklet, world-renowned specialists are featuring new insights into viral and non-viral structures used to impression gene supply, one bankruptcy being devoted to the recent box of RNA interference (RNAi). This latter strategy could be effectively utilized in renal ischemia-reperfusion damage, trauma and transplantation. within the close to destiny, gene remedy may additionally turn out to be a brand new technique to goal molecules enthusiastic about tissue harm and irritation strategies that underlie ARF. up to now, gene move has been effectively utilized in experimental glomerulonephritis and interstitial fibrosis, and experiences in greater animals are keenly awaited. additionally coated are concerns with regards to transplantation, that's the remedy of selection in lots of end-stage organ disasters. move of genes whose protein items have immunomodulatory houses have confirmed useful in treating acute and persistent graft rejection, one of many difficulties now not satisfactorily solved through present anti-rejection medicines. Gene treatment therefore may possibly develop into a truth in scientific transplantation as soon as its efficacy in better animals has been validated. final yet now not least, a potential good thing about precise gene treatment in renal melanoma or HIV-associated nephropathy is explored. protecting a large spectrum of issues, this ebook offers a necessary review of present advancements and concerns.
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Extra info for Gene Therapy for Renal Diseases and Transplantation
Rulli SJ Jr, Hibbert CS, Mirro J, Pederson T, Biswal S, Rein A: Selective and nonselective packaging of cellular RNAs in retrovirus particles. J Virol 2007;81:6623–6631. Epub 2007 Mar 28.
E. intranuclear decondensation and localization) and to minimize cytoplasmic interactions between mRNA and vectors are needed to further improve nonviral vectors. Conclusion Although a significant proportion of renal failure cases are fatal, protocols to cure the underlying renal diseases per se remain to be established. The development of gene therapy for the treatment of kidney disease requires improved gene delivery systems that can target specific renal compartments. e. nucleus and cytosol, respectively).
In designing gene therapy applications for nephrology the ideal is to target the therapeutic gene directly to the kidney and if possible express it in specific target cells. The kidney is a particularly challenging organ because it has a complex structure with functional and cellular heterogeneity. Retroviral vectors are an attractive vehicle for delivering genes to kidney cells because they insert the therapeutic gene into cellular DNA for long-term expression. Lentiviral vectors are particularly favorable due to their ability to infect nondividing differentiated cells that comprise the majority of cells in the developed adult kidney.
Gene Therapy for Renal Diseases and Transplantation by A. Benigni, G. Remuzzi, C. Ronco