1. Phase 3 trial of RNAi therapeutic givosiran for acute intermittent porphyria;Balwani;N Engl J Med,2020
2. Fazirsiran for liver disease associated with alpha(1)-antitrypsin deficiency;Strnad;N Engl J Med,2022
3. How to achieve functional cure of HBV: stopping NUCs, adding interferon or new drug development?;Wong;J Hepatol,2022
4. U.S. Food and Drug Administration. Approved cellular and gene therapy products from: https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products; last accessed 25/01/2025.
5. European Medicine Agency. Approved cellular and gene therapy products from: https://www.ema.europa.eu/en/human-regulatory/overview/advanced-therapy-medicinal-products-overview; last accessed 25/01/2025.