In More Detail…..
Patient Clinical Trials to cure FOXG1 syndrome, including drug and gene therapies
In order to achieve these goals, we are working on the following basic science and gene and drug testing initiatives. See the specific research projects we have underway, and that we are raising money to fund HERE.
MODEL FOXG1 SYNDROME IN ANIMALS AND HUMAN STEM CELLS
Create FoxG1 mutated mice models, zebrafish models, and other models. We are building a comprehensive set of animal models for all mutations (Learn more)
Create patient derived stem cells (induced pluripotent stem cells or iPSC’s) and three dimensional organoids (brains in a dish) for the gamut of mutations.
UNDERSTAND FOXG1 SYNDROME IMPACT ON ANIMAL AND HUMAN STEM CELL MODELS
Study the above models to understand the impact of FOXG1 on the brain
Answer questions such as, but not limited to, how Foxg1 impacts protein expression, transcription activity, neuronal differentiation; and how various mutations bind to DNA
Characterize the symptoms seen in animal models and stem cells arising from each mutation
Learn if FOXG1 mutations can be "fixed" postnatally
IDENTIFY AND VET GENE THERAPIES
We will test various gene therapies on animal and human stem cell models such as:
Activating / Inserting a healthy copy of the FOXG1 gene (saRNA)
Cutting down the FOXG1 gene (RNAi)
Base Swap Repair at the RNA or DNA level
Editing at the DNA level (CRISPR)
As technology advances, we will continue to research new therapies and:
learn which time points are effective for treatment
Identify successful therapies and test efficacy
FOXG1 syndrome is an excellent candidate for gene therapy. Learn why.
IDENTIFY AND VET SMALL MOLECULE AND OTHER THERAPIES
Screen existing FDA approved small molecules on identified biological pathways that can modulated
See the projects we have underway, and those we are raising money to fund, in our Research Projects page.