Pasquale Piccolo

Pasquale Piccolo

Molecular Therapy

Liver-Directed Gene Therapy




Understanding and treating rare genetic diseases is a wonderful journey and an extraordinary creative adventure that teaches us a lot about human life and health.

Additional Funding
  • EC- AAVolution - Next-generation AAV vectors for liver-directed gene therapy (2022-2026)
  • ALP, ALPHA1-FOUNDATION - Mitochondrial dysfunction in a1-antitrypsin deficiency- associated liver disease (2022-2024)
  • EASL - Promoterless liver genome editing for Progressive Familiar Intrahepatic Cholestasis type 3 (2022-2024)