Spliceosome-mediated trans-splicing: the therapeutic cut and paste - PubMed (original) (raw)
Review
. 2012 Aug;132(8):1959-66.
doi: 10.1038/jid.2012.101. Epub 2012 Apr 12.
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- PMID: 22495179
- DOI: 10.1038/jid.2012.101
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Review
Spliceosome-mediated trans-splicing: the therapeutic cut and paste
Verena Wally et al. J Invest Dermatol. 2012 Aug.
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Abstract
Spliceosome-mediated RNA trans-splicing (SMaRT) is an RNA-based technology to reprogram genes for diagnostic and therapeutic purposes. For the correction of genetic diseases, SMaRT offers several advantages over traditional gene-replacement strategies. SMaRT protocols have recently been used for in vitro phenotypic correction of a variety of genetic disorders, ranging from epidermolysis bullosa to neurodegenerative diseases. In vivo studies are currently bringing trans-splicing RNA therapy toward clinical application. In this review, we summarize the progress made toward the medical use of SMaRT and provide an outlook on its upcoming applications.
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