In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease.
Bose SK, White BM, Kashyap MV, Dave A, De Bie FR, Li H, Singh K, Menon P, Wang T, Teerdhala S, Swaminathan V, Hartman HA, Jayachandran S, Chandrasekaran P, Musunuru K, Jain R, Frank DB, Zoltick P, Peranteau WH.
Bose SK, et al. Among authors: swaminathan v.
Nat Commun. 2021 Jul 13;12(1):4291. doi: 10.1038/s41467-021-24443-8.
Nat Commun. 2021.
PMID: 34257302
Free PMC article.