Therapeutic Research for

FOXG1 Syndrome

Our goal is simple: to develop treatments that can improve the lives of people living with FOXG1 syndrome. We are pursuing three complementary research programmes that move from identifying potential therapies to testing cutting-edge genetic approaches.

1. Repurposing Existing Medicines

One of the fastest ways to find new treatments is to investigate medicines that are already approved for other conditions.

Using our zebrafish models of FOXG1 syndrome, we will test 50 FDA-approved compounds to identify drugs that can improve the sleep difficulties seen in the condition. Sleep problems are one of the most common and challenging features experienced by individuals with FOXG1 syndrome.

Any medicines that successfully improve sleep will then be tested to see whether they also improve other aspects of the condition. Because these drugs have already passed safety testing for human use, successful results could accelerate the path towards clinical trials.

2. Developing Gene-Targeted Therapies

Many cases of FOXG1 syndrome are caused by either too little or too much FOXG1 activity. We are developing a precision gene therapy approach designed to restore the correct level of FOXG1 expression.

Working with patient-derived brain organoids—three-dimensional miniature brain tissues grown from patients’ own cells—we will use CRISPR-based technology to either:

  • increase FOXG1 expression when one copy of the gene is missing or not working properly, or
  • reduce FOXG1 expression when an extra copy of the gene is present.

This research will provide an important proof of principle for a personalised treatment strategy. We are planning this work in collaboration with Professor Stephan Sanders and are exploring opportunities through their MRC Centre of Research Excellence.

3. Testing Gene Therapy in Living Models

After demonstrating that our CRISPR-based approach works in patient-derived cells, the next step is to test whether it can improve symptoms in living models of FOXG1 syndrome.

Using our zebrafish models, we will investigate whether treatment at different stages of development can restore normal brain function and behaviour. This work will help determine the best timing for future therapies and provide essential evidence before moving towards clinical studies.

Why Your Support Matters

These three projects represent a clear pathway towards new treatments for FOXG1 syndrome – from identifying existing medicines that could rapidly reach patients, to developing innovative gene therapies that address the underlying cause of the condition.

Every donation helps us move these promising discoveries closer to the families who urgently need them.