Abstract
Spliceosome-mediated RNA trans-splicing (SMaRT) is a tool that facilitates the recombination of two distinct pre-mRNA molecules. Its application for gene therapeutic purposes has been hindered by laborious procedures to identify gene-specific molecules. We have established a screening method for the identification of highly functional RNA trans-splicing molecules based on fluorescence reporters, facilitating the generation of most potent therapeutic molecules for the correction of any gene of interest.
MeSH terms
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Animals
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Base Sequence
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Cell Culture Techniques / methods
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Cell Line
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Cloning, Molecular / methods
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DNA Primers / genetics
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Flow Cytometry / methods
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Genes, Reporter
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Genetic Therapy / methods*
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Green Fluorescent Proteins / genetics
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Humans
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Luminescent Proteins / genetics
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Molecular Sequence Data
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Polymerase Chain Reaction / methods
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RNA, Messenger / genetics
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Skin / metabolism
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Skin Diseases / genetics*
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Skin Diseases / therapy
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Spliceosomes / genetics
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Trans-Splicing*
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Transfection
Substances
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DNA Primers
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Luminescent Proteins
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RNA, Messenger
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fluorescent protein 583
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Green Fluorescent Proteins