In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery - Bahal, Raman; Ali McNeer, Nicole; Quijano, Elias; Liu, Yanfeng; Sulkowski, Parker; Turchick, Audrey; Lu, Yi-Chien; Bhunia, Dinesh C.; Manna, Arunava; Greiner, Dale L.; Brehm, Michael A.; Cheng, Christopher J.; et al. (Nature communications, 2016)
Keywords: 13; 42; 42/41; 631/61/391/2308; 692/4017; Animals; beta-Globins - genetics; beta-Thalassemia - blood; beta-Thalassemia - genetics; beta-Thalassemia - therapy; Cell Line; Disease Models, Animal; DNA - administration & dosage; DNA - genetics; Gene Editing - methods; Genetic Therapy - methods; Hematopoietic Stem Cells - metabolism; Hemoglobins - analysis; Humanities and Social Sciences; Humans; Injections, Intravenous; Mice; Mice, Transgenic; multidisciplinary; Nanoparticles - administration & dosage; Peptide Nucleic Acids - administration & dosage; Peptide Nucleic Acids - genetics; Proto-Oncogene Proteins c-kit - metabolism; Science; Science (multidisciplinary); Stem Cell Factor - administration & dosage; Stem Cell Factor - metabolism