Article
Targeted correction of a thalassemia-associated beta-globin mutation induced by pseudo-complementary peptide nucleic acids.
Nucleic acids research - 1 Jun 2009
Lonkar Pallavi, Kim Ki-Hyun, Kuan Jean Y, Chin Joanna Y, Rogers Faye A, Knauert Melissa P, Kole Ryszard, Nielsen Peter E, Glazer Peter M
Abstract excerpt
Beta-thalassemia is a genetic disorder caused by mutations in the beta-globin gene. Triplex-forming oligonucleotides and triplex-forming peptide nucleic acids (PNAs) have been shown to stimulate recombination in mammalian cells via site-specific binding and creation of altered helical structures that provoke DNA repair. However, the use of these molecules for gene targeting requires homopurine tracts to...
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