New 4H leukodystrophy study to be led by RI-MUHC researcher

MRM member Dr. Geneviève Bernard of the Research Institute of the McGill University Health Centre (RI-MUHC) is partnering with the Yaya Foundation for 4H Leukodystrophy, and with Drs. Guangping Gao and Jun Xie of the University of Massachusetts Medical School to develop models and to study a potential gene therapy for 4H leukodystrophy. This rare, progressive, genetic neurological disorder primarily affects children. There is not yet a treatment or cure.

A six-figure commitment from the Yaya Foundation will enable the researchers to develop and characterize a suite of critical research tools, and develop a pre-clinical gene therapy proof of concept.

“I am excited to be partnering with Dr. Gao and the Yaya Foundation on this study,” said Dr. Bernard, a scientist in the Child Health and Human Development Program at the RI‑MUHC. “It is only with collaboration between patients, families and researchers with complementary skill sets that we will be able to drive better outcomes for these patients that I care so much about.”

“Gene therapy directly targets the underlying disease biology, which I believe has the potential to lead to transformative treatments,” said Dr. Gao.

This project has been made possible in part by grants from the Chan Zuckerberg Initiative Rare As One Project and the Tallman Family Charitable Fund.

About the Yaya Foundation for 4H Leukodystrophy

Founded by a group of parents of children with 4H leukodystrophy, the Yaya Foundation is the only organization in the world dedicated to fighting for patients and families affected by 4H leukodystrophy. For more information, please visit

Source: Health e-News, see their story here.

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