| pubmed-article:11127576 | rdf:type | pubmed:Citation | lld:pubmed |
| pubmed-article:11127576 | lifeskim:mentions | umls-concept:C0374711 | lld:lifeskim |
| pubmed-article:11127576 | lifeskim:mentions | umls-concept:C0043240 | lld:lifeskim |
| pubmed-article:11127576 | lifeskim:mentions | umls-concept:C0035696 | lld:lifeskim |
| pubmed-article:11127576 | lifeskim:mentions | umls-concept:C1413365 | lld:lifeskim |
| pubmed-article:11127576 | lifeskim:mentions | umls-concept:C0600448 | lld:lifeskim |
| pubmed-article:11127576 | lifeskim:mentions | umls-concept:C1705181 | lld:lifeskim |
| pubmed-article:11127576 | pubmed:issue | 22 | lld:pubmed |
| pubmed-article:11127576 | pubmed:dateCreated | 2000-12-20 | lld:pubmed |
| pubmed-article:11127576 | pubmed:abstractText | Most messenger RNA precursors (pre-mRNA) undergo cis-splicing in which introns are excised and the adjoining exons from a single pre-mRNA are ligated together to form mature messenger RNA. This reaction is driven by a complex known as the spliceosome. Spliceosomes can also combine sequences from two independently transcribed pre-mRNAs in a process known as trans-splicing. Spliceosome-mediated RNA trans-splicing (SMaRT) is an emerging technology in which RNA pre-therapeutic molecules (PTMs) are designed to recode a specific pre-mRNA by suppressing cis-splicing while enhancing trans-splicing between the PTM and its pre-mRNA target. This study examined the feasibility of SMaRT as a potential therapy for genetic diseases to correct mutations using cystic fibrosis (CF) as an example. We used several versions of a cystic fibrosis transmembrane conductance regulator (CFTR) mini-gene expressing mutant (deltaF508) pre-mRNA targets and tested this against a number of PTMs capable of binding to the CFTR target intron 9 and trans-splicing in the normal coding sequences for exons 10-24 (containing F508). When 293T cells were cotransfected with both constructs, they produced a trans-spliced mRNA in which normal exon 10-24 replaced mutant exon 10. To test whether SMaRT produced mature CFTR protein, proteins were immunoprecipitated from lysates of cotransfected cells and detected by Western blotting and PKA-phosphorylation. Tryptic phosphopeptide mapping confirmed the identity of CFTR. This proof-of-concept study demonstrates that exon replacement by SMaRT can repair an abnormal pre-mRNA associated with a genetic disease. | lld:pubmed |
| pubmed-article:11127576 | pubmed:grant | http://linkedlifedata.com/r... | lld:pubmed |
| pubmed-article:11127576 | pubmed:grant | http://linkedlifedata.com/r... | lld:pubmed |
| pubmed-article:11127576 | pubmed:language | eng | lld:pubmed |
| pubmed-article:11127576 | pubmed:journal | http://linkedlifedata.com/r... | lld:pubmed |
| pubmed-article:11127576 | pubmed:citationSubset | IM | lld:pubmed |
| pubmed-article:11127576 | pubmed:chemical | http://linkedlifedata.com/r... | lld:pubmed |
| pubmed-article:11127576 | pubmed:chemical | http://linkedlifedata.com/r... | lld:pubmed |
| pubmed-article:11127576 | pubmed:chemical | http://linkedlifedata.com/r... | lld:pubmed |
| pubmed-article:11127576 | pubmed:chemical | http://linkedlifedata.com/r... | lld:pubmed |
| pubmed-article:11127576 | pubmed:chemical | http://linkedlifedata.com/r... | lld:pubmed |
| pubmed-article:11127576 | pubmed:chemical | http://linkedlifedata.com/r... | lld:pubmed |
| pubmed-article:11127576 | pubmed:chemical | http://linkedlifedata.com/r... | lld:pubmed |
| pubmed-article:11127576 | pubmed:status | MEDLINE | lld:pubmed |
| pubmed-article:11127576 | pubmed:month | Nov | lld:pubmed |
| pubmed-article:11127576 | pubmed:issn | 0969-7128 | lld:pubmed |
| pubmed-article:11127576 | pubmed:author | pubmed-author:YangC CCC | lld:pubmed |
| pubmed-article:11127576 | pubmed:author | pubmed-author:CohnJ AJA | lld:pubmed |
| pubmed-article:11127576 | pubmed:author | pubmed-author:Garcia-Blanco... | lld:pubmed |
| pubmed-article:11127576 | pubmed:author | pubmed-author:KolbMM | lld:pubmed |
| pubmed-article:11127576 | pubmed:author | pubmed-author:MitchellL GLG | lld:pubmed |
| pubmed-article:11127576 | pubmed:author | pubmed-author:PuttarajuMM | lld:pubmed |
| pubmed-article:11127576 | pubmed:author | pubmed-author:MansfieldS... | lld:pubmed |
| pubmed-article:11127576 | pubmed:issnType | Print | lld:pubmed |
| pubmed-article:11127576 | pubmed:volume | 7 | lld:pubmed |
| pubmed-article:11127576 | pubmed:owner | NLM | lld:pubmed |
| pubmed-article:11127576 | pubmed:authorsComplete | Y | lld:pubmed |
| pubmed-article:11127576 | pubmed:pagination | 1885-95 | lld:pubmed |
| pubmed-article:11127576 | pubmed:dateRevised | 2007-11-14 | lld:pubmed |
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| pubmed-article:11127576 | pubmed:year | 2000 | lld:pubmed |
| pubmed-article:11127576 | pubmed:articleTitle | Repair of CFTR mRNA by spliceosome-mediated RNA trans-splicing. | lld:pubmed |
| pubmed-article:11127576 | pubmed:affiliation | Intronn, LLC, Durham, NC 27701, USA. | lld:pubmed |
| pubmed-article:11127576 | pubmed:publicationType | Journal Article | lld:pubmed |
| pubmed-article:11127576 | pubmed:publicationType | Research Support, U.S. Gov't, P.H.S. | lld:pubmed |
| pubmed-article:11127576 | pubmed:publicationType | Research Support, Non-U.S. Gov't | lld:pubmed |
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