Dr. Mingshan Xue Named 2025 Oxford-Harrington Rare Disease Scholar; Validating Project CASK-Funded Gene Therapy Research

Dr. Mingshan Xue Named 2025 Oxford-Harrington Rare Disease Scholar

In November 2025, Dr. Mingshan Xue of Baylor College of Medicine was named a 2025 Oxford-Harrington Rare Disease Scholar by the Oxford-Harrington Rare Disease Centre, one of the most selective therapeutic development honors in the world.

Dr. Xue is leading gene replacement therapy research for CASK gene disorders, funded in part by Project CASK. Project CASK identified Dr. Xue's research as a critical funding opportunity in 2023 and committed $375,000 in July 2024 to accelerate his preclinical work. His research is developing a disease-modifying treatment applicable to all CASK mutations, including both females and males with CASK-related disorders such as MICPCH and X-linked intellectual disability.

The Oxford-Harrington Rare Disease Centre selects scholars based on the scientific rigor and therapeutic potential of their work. The 2025 Scholar award validates both Dr. Xue's science and the strategic judgment of the parent-led funding that made it possible.

The strengthened preclinical data from Project CASK's investment attracted the attention of the Oxford-Harrington program.

"By identifying promising science and deploying parent-driven capital at a critical moment, we accelerated preclinical work by a leading researcher who caught the attention of one of the world's most prestigious rare disease programs," Project CASK noted in its 2025 Annual Impact Report.

CASK gene disorders affect fewer than 600 diagnosed families worldwide across 54 countries. There are currently no approved treatments. Project CASK is the largest single parent advocacy funder of CASK gene disorder research globally, having committed over $650,000 across 11 active studies.

About Dr. Mingshan Xue

Dr. Mingshan Xue is a researcher at Baylor College of Medicine. His laboratory focuses on understanding the molecular and cellular mechanisms of neurological disorders, with a focus on developing gene replacement strategies for rare neurodevelopmental conditions.

About the Oxford-Harrington Rare Disease Centre

The Oxford-Harrington Rare Disease Centre is a partnership between the University of Oxford and the Harrington Discovery Institute. The Rare Disease Scholar program supports researchers with exceptional therapeutic development potential, providing both funding and expert mentorship to accelerate the path from lab to clinic.

Read the full announcement at www.oxfordharrington.org