The Research Post-Doctoral Fellow will support basic science research projects within the Leukodystrophy Center at the Children's Hospital of Philadelphia. This is a a cutting-edge translational program that supports therapeutic development for a group of approximately 31 rare genetic disorders that affect development of the white matter of the brain, predominantly in pediatric populations. Current projects are focused on therapeutic development - using in vivo and in vitro models - for key leukodystrophyies such as Hypomyelination and Atrophy of Basal Ganglia and Cerebellum (H-ABC), Aicardi-Goutières Syndrome (AGS), and Alexander Disease.
The Leukodystrophy Center is currently seeking candidates for a post-doctoral position to focus on a research project involving development of novel therapeutic approaches using adeno-associated virus (AAV)-based gene therapy and antisense oligonucleotides (ASOs) in these target disorders. The position will include opportunities to participate in the design, optimization, and establishment of feasibility and safety of the proposed therapeutic approaches preparatory to clinical development. The laboratory, under the direction of Dr. Adeline Vanderver, MD, uses a variety of advanced scientific techniques, such as immunohistochemistry, confocal microscopy, stereotaxic surgery, and molecular biology assays. The post-doctoral fellowship includes participation in various training and career development programs to develop skills in key areas such as data management, biostatistics, manuscript preparation, etc. The post-doctoral fellow will receive direct mentorship from Dr. Vanderver and other senior scientists within the laboratory, tailed to training program within the lab will be individually tailored to prepare for their desired next career steps.
This position also involves external collaboration with other key leaders in the field of leukodystrophy. Dr. Vanderver is a co-founder of the Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN), a Rare Diseases Clinical Research Network (RDCRN) consortium funded under grant number U54NS115052 in collaboration between the National Center for Advancing Translational Sciences (NCATS) and the National Institute of Neurological Disorders and Stroke (NINDS). The post-doctoral fellow will have opportunities to participate in grant-funded research collaborations, and attend/present at various scientific meetings and seminars.