Neurology · 2021 · 11 citations · 0 references
MedicineNeurologyNeuroscienceGene TherapyMedical GeneticsNeurogeneticsGenetic DisordersHereditary NeuropathiesNeurodevelopmental DisordersNeurogenetic DisordersNeurological Gene TherapySafety Of Gene TherapyEditing For Neurological DisordersGenetic Neurodevelopmental SyndromesNeurodegenerative Disease Gene TherapyNeurodevelopmental MilestonesNeurodevelopmental Outcomes Of Growth
To evaluate the safety and efficacy of CNS-administered gene therapy for severe MPS II