Ricciardi, Adele S

In utero nanoparticle delivery for site-specific genome editing. [electronic resource] - Nature communications 06 2018 - 2481 p. digital

Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't

2041-1723

10.1038/s41467-018-04894-2 doi


Animals
DNA, Single-Stranded--administration & dosage
Disease Models, Animal
Female
Fetal Therapies--methods
Gene Editing--methods
Genetic Diseases, Inborn--genetics
Humans
Mice
Mice, Inbred C57BL
Mice, Mutant Strains
Nanoparticles--administration & dosage
Peptide Nucleic Acids--administration & dosage
Pregnancy
Safety
Targeted Gene Repair--methods
Uterus
beta-Globins--genetics
beta-Thalassemia--blood