We are seeking a postdoctoral fellow with experience in iPSC-derived neurons to study cellular and molecular mechanisms underlying the motor neuron disease spinal muscular atrophy (SMA). The long term goal of my laboratory in the Departments of Neurology and Neuroscience at Johns Hopkins University School of Medicine is to develop treatments for spinal muscular atrophies and Charcot-Marie-Tooth disease. Although 3 new gene targeting therapeutics have been approved for SMA due to SMN protein deficiency in the last 4 years, therapeutic efficacy in patients remains highly variable. It remains unknown how decreased SMN protein expression causes disease. We have recently shown that SMN expression is developmentally regulated (PMID: 31589162) and that in patients impairments of motor neuron axon development begin in utero and are followed by precipitous axonal degeneration postnatally (PMID: 33504650). We are using patient tissues, mouse models, and compartmentalized iPSC cell derived neurons together with advanced imaging techniques and biochemistry to understand the molecular mechanisms that cause these pathologies and to develop combinatorial therapeutics.
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