Lyndsey Braun

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

Jerry R. Mendell, Samiah Al-Zaidy, Richard Shell et al. · New England Journal of Medicine · 2017 · 2.3K citations · Full text

Medicine, Genetics, Neurology +16

RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

Kevin D. Foust, Xueyong Wang, Vicki L. McGovern et al. · Nature Biotechnology · 2010 · 690 citations · Full text

Medicine, Neurobiology, Neuroscience +10

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