In utero nanoparticle delivery for site-specific genome editing

• Published in: Nature communications Vol. 9; no. 1; pp. 2481 - 11
• Main Authors: 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, Ly, Danith H., Stitelman, David H., Glazer, Peter M., Saltzman, W. Mark
• Format: Journal Article
• Language: English
• Published: London Nature Publishing Group UK 26.06.2018; Nature Publishing Group; Nature Portfolio
• Subjects: 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

Genetic diseases can be diagnosed early during pregnancy, but many monogenic disorders continue to cause considerable neonatal and pediatric morbidity and mortality. Early intervention through intrauterine gene editing, however, could correct the genetic defect, potentially allowing for normal organ...