AAV Gene Therapy Vectors: Design and Production
Adeno-Associated Virus (AAV) is the leading viral vector for gene therapy. Safe, effective, but with integration and immune response risks.
Vector Design
Capsid Engineering
Production & Titer
Risks ⚠️
Dosing: 10^12 - 10^14 vg/kg (patient weight-based)
Related Chronicles: Synthetic Blood Contamination (2032)
FDA Approved: Luxturna (retina), Zolgensma (SMA)