New Gene Therapy Vehicle for Brain Diseases
Introduction
Researchers have developed a new way to deliver genes to the brain using a specially engineered virus. This new method shows promise for treating various brain disorders.
By Alessandra DiCorato May 16, 2024
Overview
What They Did
- Scientists at the Broad Institute of MIT and Harvard created a modified adeno-associated virus (AAV).
- This AAV can cross the blood-brain barrier, a protective shield that separates the brain from the bloodstream.
- The virus successfully delivered genes to the brain in mice modified to have human-like blood-brain barriers.
Why It Matters
- Traditional AAVs have struggled to cross the blood-brain barrier, making it hard to treat brain diseases.
- This new AAV could be used to treat severe genetic brain disorders like Parkinson’s disease, Gaucher’s disease, and various neurodegenerative diseases.
How It Works
The Science Behind It
- The new AAV binds to the human transferrin receptor, a protein that helps substances cross the blood-brain barrier.
- In tests, this virus reached high levels in the brains of mice with human-like blood-brain barriers.
Testing and Results
- When injected into the bloodstream, the new AAV crosses into the brain much more effectively than current FDA-approved AAVs.
- It delivered the GBA1 gene, linked to several brain disorders, to many brain cells.
Potential Applications
- The new AAV could treat various brain diseases by delivering healthy genes to replace defective ones.
- It might also be effective for disorders like Rett syndrome, SHANK3 deficiency, and Huntington’s disease.
Future Directions
Next Steps
- Researchers aim to improve the efficiency of this gene delivery method.
- They hope to reduce the virus’s accumulation in the liver and prevent it from being neutralized by antibodies.
Real-World Impact
- A biotech company, Apertura Gene Therapy, is developing new treatments using this AAV.
- This innovation could significantly improve the lives of patients with brain disorders.
Glossary
- Adeno-Associated Virus (AAV): A small virus that delivers genes to cells.
- Blood-Brain Barrier: A selective barrier that protects the brain from harmful substances in the blood.
- Gene Therapy: A technique that uses genes to treat or prevent disease.
- Transferrin Receptor: A protein that helps transport substances across the blood-brain barrier.
- GBA1 Gene: A gene linked to Gaucher’s disease, Parkinson’s disease, and Lewy body dementia.
Credits
- Author: Alessandra DiCorato
- Research Team: Ben Deverman, Ken Chan, Qin Huang, Jason Wu, and others at the Broad Institute of MIT and Harvard
- Funding: Apertura Gene Therapy, National Institutes of Health Common Fund, National Institute of Neurological Disorders and Stroke, Stanley Center for Psychiatric Research