Combination Hematopoietic Stem Cell Transplantation and Intravenous AAVrh10-Mediated Gene Therapy in a Canine Model Proves Pivotal for Translation of Krabbe Disease Therapy.

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Investigators evaluated hematopoietic stem cell transplantation (HSCT), intravenous (IV) AAVrh10 gene therapy, and combination HSCT + IV AAVrh10 in the canine model of globoid cell leukodystrophy.
[Molecular Therapy]
AbstractGraphical Abstract