Zhao, Q., Peng, H., Ma, Y. et al. In vivo applications and toxicities of AAV-based gene therapies in rare diseases. Orphanet J Rare Dis 20, 368 (2025). doi:10.1186/s13023-025-03893-z
As of early 2024, only eight AAV-based gene therapy drugs have been approved.AAV-based gene therapies have revolutionized treatment for rare diseases; however, addressing toxicity and improving long-term efficacy remain key challenges.
In vivo applications and toxicities of AAV-based gene therapies in rare diseases