Modulating the Expression of Disease Genes with RNA-Based Therapy.

@article{citeulike:1451417, abstract = {Conventional gene therapy has focused largely on gene replacement in target cells. However, progress from basic research to the clinic has been slow for reasons relating principally to the challenges of heterologous DNA delivery and regulation in vivo. Alternative approaches targeting RNA have the potential to circumvent some of these difficulties, particularly as the active therapeutic molecules are usually short oligonucleotides and the target gene transcript is under endogenous regulation. RNA-based strategies offer a series of novel therapeutic applications, including altered processing of the target pre-mRNA transcript, reprogramming of genetic defects through mRNA repair, and the targeted silencing of allele- or isoform-specific gene transcripts. This review examines the potential of RNA therapeutics, focusing on antisense oligonucleotide modification of pre-mRNA splicing, methods for pre-mRNA trans-splicing, and the isoform- and allele-specific applications of RNA interference.}, author = {Wood, Matthew and Yin, Haifang and McClorey, Graham }, citeulike-article-id = {1451417}, doi = {http://dx.doi.org/10.1371/journal.pgen.0030109}, issn = {1553-7404}, journal = {PLoS Genet}, month = {June}, number = {6}, posted-at = {2007-07-24 20:34:12}, priority = {2}, title = {Modulating the Expression of Disease Genes with RNA-Based Therapy.}, url = {http://dx.doi.org/10.1371/journal.pgen.0030109}, volume = {3}, year = {2007} }

TY - JOUR ID - citeulike:1451417 L3 - citeulike-article-id:1451417 N2 - Conventional gene therapy has focused largely on gene replacement in target cells. However, progress from basic research to the clinic has been slow for reasons relating principally to the challenges of heterologous DNA delivery and regulation in vivo. Alternative approaches targeting RNA have the potential to circumvent some of these difficulties, particularly as the active therapeutic molecules are usually short oligonucleotides and the target gene transcript is under endogenous regulation. RNA-based strategies offer a series of novel therapeutic applications, including altered processing of the target pre-mRNA transcript, reprogramming of genetic defects through mRNA repair, and the targeted silencing of allele- or isoform-specific gene transcripts. This review examines the potential of RNA therapeutics, focusing on antisense oligonucleotide modification of pre-mRNA splicing, methods for pre-mRNA trans-splicing, and the isoform- and allele-specific applications of RNA interference. IS - 6 JF - PLoS Genet SN - 1553-7404 TI - Modulating the Expression of Disease Genes with RNA-Based Therapy. VL - 3 AU - Wood, Matthew AU - Yin, Haifang AU - McClorey, Graham PY - 2007/06/29/ UR - http://dx.doi.org/10.1371/journal.pgen.0030109 ER -