Leah Schmelzer

Microglia Induce Motor Neuron Death via the Classical NF-κB Pathway in Amyotrophic Lateral Sclerosis

Laura Ferraiuolo, Amanda Haidet-Phillips, Leah Schmelzer et al. · Neuron · 2014 · 636 citations

Life Sciences, Neurobiology, Neuroscience +16

Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders

Adam K. Bevan, Sandra Duqué, Kevin D. Foust et al. · Molecular Therapy · 2011 · 331 citations · Full text

Pharmacy, Neurobiology, Neuroscience +17

Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates

Kathrin Meyer, Laura Ferraiuolo, Leah Schmelzer et al. · Molecular Therapy · 2014 · 280 citations · Full text

Medicine, Neurology, Neuroscience +17

Early heart failure in the SMNΔ7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery

Adam K. Bevan, Kirk R. Hutchinson, Kevin D. Foust et al. · Human Molecular Genetics · 2010 · 203 citations · Full text

Medicine, Cardiology, Neurology +17

A large animal model of spinal muscular atrophy and correction of phenotype

Sandra Duqué, W. David Arnold, Philipp Odermatt et al. · Annals of Neurology · 2014 · 133 citations · Full text

Medicine, Neurology, Neuroscience +17