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Targeted correction of a thalassemia-associated β-globin mutation induced by pseudo-complementary peptide nucleic acids
β-Thalassemia is a genetic disorder caused by mutations in the β-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 prov...
Autores principales: | 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. |
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Formato: | Texto |
Lenguaje: | English |
Publicado: |
Oxford University Press
2009
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2699504/ https://www.ncbi.nlm.nih.gov/pubmed/19364810 http://dx.doi.org/10.1093/nar/gkp217 |
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