The Wiedemann-Steiner Syndrome Grant Program

The Wiedemann-Steiner Syndrome Grant Program 2025, funded by the Wiedemann-Steiner Syndrome Foundation, offers 2-year grants to advance research on the rare genetic disorder, Wiedemann-Steiner Syndrome. The program aims to award at least three grants of $90,000 each for projects focusing on patient-derived cell models and Natural History Studies. These initiatives seek to enhance understanding of the syndrome and improve therapeutic strategies for affected individuals.

The Wiedemann-Steiner Syndrome Grant Program 2025
The Wiedemann-Steiner Syndrome Foundation will provide 2-year grants to support research related to Wiedemann-Steiner Syndrome. At least 3 awards will be granted.
Background
Wiedemann-Steiner Syndrome (WSS) is a rare genetic disorder resulting from mutations in the MLL gene (also known as KMT2A) on the long arm of chromosome 11. The syndrome’s genetic underpinnings were clarified in 2012 by a group of researchers led by Dr. Wendy Jones. The gene encodes a histone methyl transferase which helps modify the expression of many other genes. The condition is autosomal dominant, and in most cases, the mutation occurred de novo. From an unpublished survey of 76 WSS families, the symptom determined to be of “Very High Impact” by most families, was disruptive behaviors. This included aggressive behaviors, impulsivity, and behaviors outside of age-appropriate societal norms and the most important therapeutic goals were to reduce these behaviors and improve cognitive functioning.
The Orphan Disease Center, in collaboration with the WSS Foundation, is seeking grant applications that aim to further progress the understanding of the disease:
Two $90,000 grants to support the development and characterization, for the advancement toward therapeutic goal, of patient-derived cell models of WSS Syndrome (ex. iPSCs, neurons, reporter lines). These cells model will be deposited into a publicly accessible biobank. One $90,000 grant to support a Natural History Study. Proposals for this grant should focus on understanding the progression of the condition over time, often to support product development, especially for rare diseases. These studies are observational and aim to identify factors that influence disease development and outcomes.