Gene therapy replaces, inactivates, or introduces genes to treat or prevent disease. For patients considering or researching gene therapy, understanding how it works is the first step.
Types of Gene Therapy
- Gene replacement: Introducing a functional copy of a defective gene
- Gene editing: Correcting mutations directly in the genome (CRISPR)
- Gene silencing: Turning off genes that cause disease (RNA interference)
- Cell therapy: Modifying cells outside the body and reintroducing them
Delivery Methods
Getting therapeutic genes into cells requires delivery vehicles called vectors. Viral vectors (AAV, lentivirus) are most common, but lipid nanoparticles are emerging as a non-viral alternative.