Vanderver Laboratory Research Overview

Preclinical research projects in the Vanderver Lab seek to understand the underlying molecular mechanisms that give rise to the molecular mechanisms of disease for known leukodystrophies. This is accomplished by using advanced experimental techniques in conjunction with representative in vitro and in vivo models for select leukodystrophies such as Aicardi-Goutières Syndrome (AGS) and TUBB4A-related leukodystrophy. The results of this research will be used to define molecular targets, delivery mechanisms, and biomarkers for future clinical trials and approved therapies.

The Myelin Disorders Biorepository Project (MDBP) was founded by Dr. Adeline Vanderver over a decade ago and is currently one of the world's largest leukodystrophy biorepositories. With over 2,000 affected individuals enrolled, it houses extensively curated clinical information, as well as biological samples, radiology images, and genetic sequencing data, for a wide range of leukodystrophies and related disorders affecting the white matter of the brain. It also provides infrastructural and regulatory support to the external research sites participating in the Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN).

With support from a dedicated team of medical providers, research coordinators, regulatory experts, and technicians, the Vanderver Lab has been involved in a variety of clinical studies, including Investigator-Initiated Trials, Sponsor-Initiated Trials, as well as Expanded Access or "Compassionate Use" Programs.

The Vanderver Lab has contributed to the discovery of the genes responsible for causing various leukodystrophies, and it continues to partner with some of the world's leading diagnostic laboratories to leverage the power of next generation sequencing (NGS) techniques to identify new candidate genes and help individual families navigate the diagnostic odyssey.

The Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN) is a consortium of institutions, clinicians, scientists, and patient advocacy leaders working together to promote advances in the diagnosis and treatment of leukodystrophies. Learn more about the consortium's current research projects on their website.