Article
Correction of a splice-site mutation in the beta-globin gene stimulated by triplex-forming peptide nucleic acids.
Proceedings of the National Academy of Sciences of the United States of America - 9 Sept 2008
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
Abstract excerpt
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 cells via site-specific binding and creation of altered helical structures that provoke DNA repair. We have designed a...
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