Gene targeting

... vary across alleles or even across species. Part of my days is spent targeting Morpholino antisense molecules for gene knockdowns. Sometimes investigators want a single molecules that can be used to knock ...

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I expect that AVI BioPharma http://www.avibio.com will start systemic clinical trials of their peptide-conjugated oligos targeting dystrophin for treatment of Duchenne muscular dystrophy within a year. Their trials with bare Morpholinos have been ongoing in London. http://clinicaltrials.gov/ct2/show/NCT00159250 ...

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... AE. Inhibition of norovirus replication by morpholino oligomers targeting the 5'-end of the genome. Virology. 2008 Sep 8. [Epub ahead of ... systemic muscle and cardiac dystrophin expression and function. Hum Mol Genet. 2008 Sep 10. [Epub ahead of print] Wu B, Moulton HM, Iversen PL, Jiang ...

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... for the knockout and breed together animals that carry the knocked-out gene to establish a true-breeding line. This can take a year. Knockdowns ... more convenient. You put in an antisense oligo or double-stranded RNA targeting the gene you want to knock down. In adult animals, you need a systemic ...

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... a simple version of an mRNA rescue experiment, the coding sequence of a gene is fused to a different 5'-UTR and the mRNA is coinjected with the Morpholino targeting the gene with its wild-type 5'-UTR. Ideally, transcription of the rescue mRNA replaces ...

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