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Using 5′-PTMs to repair mutant β-globin transcripts

Trans-splicing has been used to repair mutant RNA transcripts via competition for the spliceosome using pre-trans-splicing molecules, or “PTMs.” Previous studies have demonstrated that functional PTMs can be designed for either 3′- or 5′-exon replacement, with a vast majority of the work to date foc...

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Detalhes bibliográficos
Main Authors: Kierlin-Duncan, Monique N., Sullenger, Bruce A.
Formato: Artigo
Idioma:Inglês
Publicado em: Cold Spring Harbor Laboratory Press 2007
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC1924905/
https://ncbi.nlm.nih.gov/pubmed/17556711
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1261/rna.525607
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