Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease
Yedda Li, Christopher A. Miller, Lauren Shea, Xuntian Jiang, Miguel A. Guzmán, Randy J. Chandler, Sai Mukund Ramakrishnan, Stephanie Smith, Charles P. Venditti, Carole Vogler, Daniel S. Ory, Timothy J. Ley, Mark S. Sands
Topics & Concepts
Krabbe diseaseToxicityMedicineTerm (time)DiseasePharmacologyInternal medicineLeukodystrophyQuantum mechanicsPhysicsLysosomal Storage Disorders ResearchTrypanosoma species research and implicationsStudies on Chitinases and Chitosanases