Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy.
2016
Armbruster, N., et al. (2016).
Mol Ther Methods Clin Dev 3: 16060.
2016
Armbruster, N., et al. (2016).
Mol Ther Methods Clin Dev 3: 16060.