Correction of a splice-site mutation in the beta-globin gene stimulated by triplex-forming peptide nucleic acids

Splice-site mutations in the beta-globin gene can lead to aberrant transcripts and decreased functional beta-globin, causing beta-thalassemia. Triplex-forming DNA oligonucleotides (TFOs) and peptide nucleic acids (PNAs) have been shown to stimulate recombination in reporter gene loci in mammalian ce...

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Hlavní autoři: Chin, Joanna Y., Kuan, Jean Y., Lonkar, Pallavi S., Krause, Diane S., Seidman, Michael M., Peterson, Kenneth R., Nielsen, Peter E., Kole, Ryszard, Glazer, Peter M.
Médium: Artigo
Jazyk:Inglês
Vydáno: National Academy of Sciences 2008
Témata:
Biological Sciences
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC2533221/
https://ncbi.nlm.nih.gov/pubmed/18757759
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0711793105
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