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2. Combination hematopoietic stem cell transplantation and intravenous AAVrh10-mediated gene therapy in a canine model proves pivotal for translation of Krabbe disease therapy.

4. The IAP antagonist birinapant enhances chimeric antigen receptor T cell therapy for glioblastoma by overcoming antigen heterogeneity

5. Pathology of macrophage activation syndrome in humanized NSGS mice

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