GeneticsNature CommunicationsPeer-reviewed
Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I
Natalia Gomez‐Ospina, Samantha G. Scharenberg, Nathalie Mostrel…·Matthew Hebden Porteus
This study uses CRISPR/Cas9 to edit hematopoietic stem cells to express iduronidase, correcting Mucopolysaccharidosis type I in mice.
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