Publication | Open Access

Direct central nervous system delivery provides enhanced protection following vector mediated gene replacement in a severe model of Spinal Muscular Atrophy

Regenerative MedicineSevere ModelNeuroregenerationSpinal Cord InjurySpinal Muscular AtrophyNeurologyGene DeliveryBiomedical EngineeringGene ReplacementNeural Tissue EngineeringMedicineGene VectorGene Transfer