The Angelman Syndrome Alliance (ASA) is proud to announce the recipients of its 2026 Scientific Research Grants.
This year’s call for proposals attracted exceptionally high-quality research projects, reflecting the growing momentum, innovation, and dedication within the Angelman syndrome research community.
The final funding decision was made through a vote by ASA’s 12 member organisations, ensuring a fair, transparent, and community-driven process.
The ASA is pleased to announce the following projects selected for funding in 2026:
2026 ASA Research Grant Recipients
Dr. Carmen De Caro
Project: From Diet to Brain: Food Supplementation and Microbiota-Linked Biomarkers in Angelman Syndrome (AS)
Dr. Carmen De Caro’s project explores how the gut microbiome may influence brain function and symptoms in Angelman syndrome through the “microbiota–gut–brain axis.”
Recent research suggests that gut bacteria can play an important role in neurological and neurodevelopmental conditions. Preliminary findings from Dr. De Caro’s team identified specific alterations in the gut microbiota of individuals with Angelman syndrome, including increased pro-inflammatory bacteria and reduced levels of beneficial compounds important for intestinal and brain health.
This study will investigate whether targeted probiotic supplementation can help restore microbial balance and positively influence biological markers linked to brain function, inflammation, sleep, gastrointestinal symptoms, and behaviour.
The project will involve a 12-week placebo-controlled clinical study with individuals living with Angelman syndrome and aims to identify measurable biomarkers associated with improvements in health and neurological function.
By exploring safe, nutrition-based therapeutic approaches, this research could open new avenues for supportive treatments in Angelman syndrome and contribute to the development of more personalised care strategies.
Prof. Ype Elgersma
Project: Understanding UBE3A Protein Homeostasis: Controlling UBE3A Protein Levels in the Brain
Prof. Ype Elgersma’s project focuses on understanding how the brain regulates levels of the UBE3A protein, the key protein involved in Angelman syndrome.
UBE3A plays a critical role in maintaining healthy brain function by helping cells remove proteins that are no longer needed. Maintaining the right balance of UBE3A is essential: too little causes Angelman syndrome, while too much is associated with Dup15q syndrome.
This research investigates how UBE3A regulates its own breakdown and stability within brain cells. Recent discoveries from Prof. Elgersma’s team identified rare mutations that disrupt this self-regulation process, leading to severe forms of Angelman syndrome associated with epilepsy.
By studying these mutations, the project aims to better understand how UBE3A levels are controlled in the brain and why disruptions can lead to severe neurological symptoms.
Ultimately, this work could help guide the development of future therapies designed to precisely restore healthy UBE3A levels in individuals with Angelman syndrome and related neurodevelopmental disorders.
Prof. Ben Philpot
Project: Development of Next-Generation, Peripherally-Administered ASOs to Improve Safety and Biodistribution for the Treatment of Angelman Syndrome
Prof. Ben Philpot’s project aims to improve the delivery and effectiveness of antisense oligonucleotide (ASO) therapies for Angelman syndrome.
Current ASO approaches seek to reactivate the silent paternal copy of the UBE3A gene in the brain, but existing treatments require invasive spinal injections and can have limited distribution throughout the brain.
This project will test a new method of delivering ASOs through the bloodstream using an innovative transport system designed to reach the brain more effectively and evenly.
Using advanced mouse models, the research team will compare current ASO approaches with this next-generation delivery strategy in order to improve safety, accessibility, and therapeutic effectiveness.
By improving the distribution of UBE3A reactivation across the brain, this research has the potential to significantly enhance treatment outcomes for individuals living with Angelman syndrome.
A Remarkable Year for Angelman Syndrome Research
The ASA would like to sincerely thank all researchers and clinicians who submitted proposals this year. The scientific quality, creativity, and commitment demonstrated across all applications made the selection process particularly challenging.
Every project submitted represents important work toward improving understanding, treatment, and quality of life for people living with Angelman syndrome. We are deeply grateful to everyone contributing to advancing research in our community.
We would also like to warmly thank the members of the ASA Scientific Advisory Board for their professionalism, expertise, and rigorous scientific evaluations throughout the process. Their dedication and scientific excellence are invaluable to the Angelman syndrome community. We extend a special thank you to Prof. Hanoch Kaphzan for his leadership, responsiveness, and commitment as Head of the Scientific Advisory Board.
Powered Entirely by Families and Patient Organisations
The Angelman Syndrome Alliance is unique in that all funding for these research grants comes directly from its 12 member organisations. ASA brings together resources from national Angelman syndrome patient organisations across countries in order to maximise the impact of research funding and support high-quality international scientific projects.
Importantly, ASA is a 100% volunteer-run organisation. Every aspect of the alliance — including its Board’s operational work — is carried out by parents or family members of individuals living with Angelman syndrome.
This collective commitment reflects the strength, determination, and hope of the Angelman community worldwide.
Together, we continue working toward a brighter future for all individuals and families affected by Angelman syndrome.