What is personalized gene therapy?

Personalized gene therapy is a type of treatment that uses a patient’s own genetic information (or the specific genetic mutation causing their disease) to design a therapy tailored just for them. It’s also sometimes called precision gene therapy or customized gene therapy.

How could research on Miller’s case help others?

Pursuing personalized gene therapy for Miller doesn’t just help her—it can:
🌱 Build a foundation for future therapies
📚 Create scientific knowledge about the disease
🤝 Help other families by sharing data and insights
⚡️ Accelerate regulatory and clinical pathways

What is ZMYND8 and what is a neurodevelopmental disorder?

ZMYND8 is a gene involved in chromatin remodeling and gene expression, and mutations in it have been linked to neurodevelopmental disorders (often causing intellectual disability, developmental delays, hypotonia, and sometimes seizures). Since ZMYND8 is an ultra-rare disorder, there’s little existing research or therapies.

What are the steps in the process?

To design, test, and administer a personalized gene therapy, the process would entail:

• gene sequencing

• disease modeling

• preclinical studies

• regulatory filings

• a single-patient expanded access trial (administering the therapy)