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AAV Gene Therapy for Neurodegeneration: Niemann Pick Type C1 disease

National Human Genome Research Institute, Bethesda, MD and surrounding area

Position Description:  
A postdoctoral position is available to develop pre-clinical data needed to use gene therapy to treat Niemann Pick Type C1 (NPC1) disease, a lysosomal storage disorder.  This position will extend and develop previously published proof-of-principle studies demonstrating feasibility of AAV gene therapy as a treatment for NPC1: "Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1" Chandler et al.,  Human Molecular Genetics, 2017, Vol. 26, No. 1 52-64 .  The successful applicant will work with an expert in gene therapy, Charles Venditti ( and an expert in NPC1 disease, Bill Pavan (
The project will involve NPC1 AAV vector design and the testing of novel neurotrophic AAV capsids in mouse models of NPC1, delivered by various routes. The resources include full support for animal studies, vector purification, molecular biological and biochemical assays and other needed support through the NHGRI. In addition, the project may include opportunities to interact with regulatory agencies and a biotech partner.

Applicants should have received an MD, Ph.D. or MD, PhD within the last two years. Annual stipend will be commensurate with experience and training. Applicants with experience using mouse models, with mouse neuroscience and/or gene therapy is preferred.
To Apply:
Interested applicants are encouraged to submit a cover letter with a description of career goals, curriculum vitae with publications and contact information of 3 references to Dr. Bill Pavan
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