Oligodendrocyte-targeted adeno-associated virus gene therapy for Canavan disease in children: a phase 1/2 trial
P. Leone, Robert M. Lober, Jeremy S. Francis, Olga Borodovitsyna, Kim M. Cecil, David Shera, Christopher G. Janson
Topics & Concepts
Adverse effectLeukodystrophyGenetic enhancementMedicineInterim analysisDosingWhite matterDiseaseInterimDemyelinating diseaseCerebrospinal fluidClinical trialInternal medicineVector (molecular biology)Multiple sclerosisPharmacologyVirusImmunologyBioinformaticsRecombinant DNACentral nervous system diseaseGene deliveryPhases of clinical researchOncologyExtracellularTropismPathologyCardiac Structural Anomalies and RepairNeurogenetic and Muscular Disorders ResearchFetal and Pediatric Neurological Disorders