In utero nanoparticle delivery for site-specific genome editing - Ricciardi, Adele S.; Bahal, Raman; Farrelly, James S.; Quijano, Elias; Bianchi, Anthony H.; Luks, Valerie L.; Putman, Rachael; López-Giráldez, Francesc; Coşkun, Süleyman; Song, Eric; Liu, Yanfeng; Hsieh, Wei-Che; et al. (Nature communications, 2018)
Keywords: 13/31; 13/51; 45/22; 45/23; 45/41; 45/77; 631/1647/1511; 631/61/201/2110; 631/61/350/354; 631/61/391/2308; 64/110; 64/60; Animals; beta-Globins - genetics; beta-Thalassemia - blood; beta-Thalassemia - genetics; beta-Thalassemia - therapy; Disease Models, Animal; Disorders; DNA, Single-Stranded - administration & dosage; DNA, Single-Stranded - genetics; Female; Fetal Therapies - methods; Fetuses; Gene Editing - methods; Genetic Diseases, Inborn - genetics; Genetic Diseases, Inborn - therapy; Genetic modification; Genome editing; Genomes; Hemoglobin; Homology; Humanities and Social Sciences; Humans; Intravenous administration; Mice; Mice, Inbred C57BL; Mice, Mutant Strains; Morbidity; multidisciplinary; Mutation; Nanoparticles; Nanoparticles - administration & dosage; Neonates; Nucleic acids; Peptide nucleic acids; Peptide Nucleic Acids - administration & dosage; Peptide Nucleic Acids - genetics; Pregnancy; Safety; Science; Science (multidisciplinary); Splenomegaly; Survival; Target detection; Targeted Gene Repair - methods; Thalassemia; Uterus