Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I
Natalia Gomez‐Ospina, Samantha G. Scharenberg, Nathalie Mostrel, Rasmus Otkjær Bak, Sruthi Mantri, Rolen M. Quadros, Channabasavaiah B. Gurumurthy, Ciaran M. Lee, Gang Bao, Carlos Jose Suarez, Shaukat Ali Khan, Kazuki Sawamoto, Shunji Tomatsu, Nitin Raj, Laura Donatella Attardi, , Matthew Hebden Porteus
This study uses CRISPR/Cas9 to edit hematopoietic stem cells to express iduronidase, correcting Mucopolysaccharidosis type I in mice.
Nature Communications · Sep 6, 2019Peer-reviewedGenetics