PRJNA725910
In utero split AAV9 adenine base editing corrects the multi-organ pathology in a lethal lysosomal storage disease
Mucopolysaccharidosis Type I is a lysosomal storage disease affecting multiple organs, often leading to early postnatal cardiopulmonary demise. We assessed in utero adeno-associated virus delivery of base editor targeting the mutation in the MPS-IH mouse. These are the next-generation sequencing files associated with publication evaluating genetic correction.