Post-doctoral Fellow

The Section on Molecular Dysmorphology in the intramural research program of the Eunice Kennedy Shriver National Institute of Child Health and Human Development is a combined Clinical and Basic research team that is looking for a Postdoctoral Fellow with experience in gene therapy who can complement and advance our efforts to develop therapies for rare genetic disorders. Understanding the neuropathology and identifying potential therapeutic interventions for Niemann-Pick type C (NPC) disease is currently the major focus of our basic/translational research group. NPC is a progressive neurodegenerative disorder that primarily affects children. We utilize multiple preclinical models including neurons derived from induced pluripotent stem cells and mouse models. Advanced sequencing technologies, including single nuclear RNAseq and spatial transcriptomics, will be used to gain insight into pathological mechanisms. High throughput drug and genome-wide sgRNA screens are helping elucidate potential drugs or druggable pathways. Proteomic and lipidomic studies are being used to identify biomarkers important for facilitating clinical trials. We are now looking to expand our AAV9-mediated gene therapy efforts with the goal of advancing to a clinical trial. An individual with experience in gene therapy and a desire to contribute to the development of therapies for a rare disease would be an impactful addition to our team. Specifically, in vitro work with AAV9 transduction in different cell types, delivery of AAV9 constructs to mice via retro-orbital or direct CNS routes, behavioral analyses of mice, tissue collection, and final processing of tissues for readout measures using western blot, immunofluorescence staining, and droplet digital PCR are the main techniques to be carried out.

Candidates with a PhD in Biological Sciences, or a related field and less than 5 years of prior postdoctoral experience are encouraged to apply. Bench experience with gene therapy and preclinical disease models is required. Strong knowledge of computational methods to study biochemistry/biology is a plus. Applicants MUST be a US citizen or a permanent resident.

U.S. citizens and permanent residents are eligible to apply. NIH welcomes foreign nationals with the exception of individuals from this list.