This year marks a significant milestone in our mission to advance research into STXBP1-related disorders. With over $500,000 in combined funding, we’re powering groundbreaking organoid and rat chimera studies that will deepen our understanding of the disorder and accelerate the path toward effective treatments.
A Strategic Grant to Advance Brain Research
Earlier this year, Rafa’s Moonshot awarded a $100,000 grant to Dr. Omer Revah, Principal Investigator of the Revah Lab at The Hebrew University of Jerusalem (HUJI).
Dr. Revah and his team are pioneering research using human cortical organoids, three-dimensional brain-like structures grown from stem cells, to study early brain development and explore how new therapies may affect it.
From Seed Funding to Major Support
The project quickly gained momentum. Just months later, the Revah Lab secured an additional $400000 from the Israel Science Foundation (ISF) for a complementary study.
Together, these awards now represent over $500,000 in dedicated funding for organoid and transplant model research in STXBP1.
As Dr. Revah described:
“We’re grateful to begin a new collaboration with Rafa’s Moonshot, focused on better understanding and ultimately helping treat STXBP1-related disorders. We’ve also recently received support from the ISF to study STXBP1 haploinsufficiency using human cortical organoid and transplant models. We will explore how early disruptions in cortical development may lead to circuit-level changes later in life.”
Rat Chimera Models: A Window Into the Living Brain
One particularly exciting aspect of the Revah Lab’s work involves rat chimera models.
In this innovative approach, human cortical organoids are transplanted into the brains of newborn rats. This allows scientists to study human neurons as they integrate into a living brain, form connections, and respond to sensory input — offering insights that cannot be achieved in a dish alone.
For STXBP1 research, this model is especially powerful, as it enables the study of how early genetic disruptions may lead to changes in brain circuits and function over time.
The Research Goals and Impact
This combined funding will enable the Revah Lab to:
- Test and evaluate the potential effects of different therapeutic approaches
- Model the developing human brain using advanced organoid technology
- Understand how early disruptions in cortical development might affect brain circuitry later in life
- Leverage rat chimera models to study human neuron behavior in a living brain
Why This Matters for STXBP1 Families
For families living with STXBP1-related disorders, this is more than a scientific milestone. It’s momentum. Every step forward in research brings us closer to effective treatments and improved quality of life.
We are deeply grateful to our scientific community for believing in this mission. Your support is helping transform hope into tangible progress.
Together, we are accelerating the path to breakthroughs that can change lives. For STXBP1 research, this model is especially powerful, as it enables the study of how early genetic disruptions may lead to changes in brain circuits and function over time.
