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Vanderver Laboratory
The Vanderver Lab’s translational research model is aimed at developing a robust understanding of leukodystrophies and other heritable conditions affecting the white matter of the brain.
The Vanderver Lab is a founding member of the Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN), a Rare Diseases Clinical Research Network (RDCRN) consortium of scientists, industry stakeholders, and patient advocacy leaders working together to promote advances in the diagnosis and treatment of leukodystrophies.
In parallel to these approaches, the Vanderver Lab works closely with a diverse group of international stakeholders to promote disease awareness and education, advocate for the adoption of universal newborn screening and early diagnostic programs, and establish clinical guidelines to support the short- and long-term care of individuals living with leukodystrophies.
Meet the Vanderver team in their Lab Life video. Discoveries in the Vanderver Lab have led to meaningful advances in how we approach and treat leukodystrophies and other white matter disorders.
Research Highlights
Research in the Vanderver Lab focuses primarily on:
- Characterizing previously unclassified leukodystrophies and uncovering their underlying genetic causes
- Exploring the molecular mechanisms of disease using advanced in vitro and in vivo models
- Assessing the validity of advanced genetic sequencing techniques in the diagnosis of these disorders
- Leveraging large-scale biomarker and natural history studies to facilitate development of therapeutic trials and, eventually, approved clinical therapies for select leukodystrophies.
Leader
Adeline L. Vanderver, MD
Program Director, Leukodystrophy Center of Excellence
Using translational approaches that encompass genomic studies, biomarker development, disease modeling, natural history studies, and clinical trials, Dr. Vanderver seeks to improve the quality of life and lifespan of individuals living with leukodystrophies or heritable disorder of myelin. She leads the Leukodystrophy Center of Excellence at Children's Hospital of Philadelphia.