MedicineBiology
The future of gene therapy for inborn errors of metabolism: insights into liver-targeted innovations
Gloria González-Aseguinolaza, Pasquale Piccolo
Abstract
1. Liver-directed gene therapy has progressed from conceptual promise to clinical reality for several inborn errors of metabolism (IEMs). The liver is a particularly attractive target because it ho...
Citation format
GONZÁLEZ-ASEGUINOLAZA, Gloria; PICCOLO, Pasquale. The future of gene therapy for inborn errors of metabolism: Insights into liver-targeted innovations. EXPERT OPINION ON BIOLOGICAL THERAPY, 2026, 26(1): 11–14.