Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies
2023; American Society for Clinical Investigation; Volume: 134; Issue: 1 Linguagem: Inglês
10.1172/jci173510
ISSN1558-8238
AutoresStephanie M. Salabarria, Manuela Corti, Kirsten E. Coleman, Megan B. Wichman, Julie Berthy, Precilla D’Souza, Cynthia J. Tifft, Roland W. Herzog, Melissa E. Elder, Lawrence R. Shoemaker, Carmen Leon‐Astudillo, Fatemeh Tavakkoli, David H. Kirn, Jonathan D. Schwartz, Barry J. Byrne,
Tópico(s)Virus-based gene therapy research
ResumoBACKGROUND. Systemic administration of Adeno-associated virus (AAV) can trigger life-threatening inflammatory responses including thrombotic microangiopathy (TMA), acute kidney injury due to atypical hemolytic uremic syndrome (aHUS)-like complement activation, immune-mediated myocardial inflammation and hepatic toxicity.
Referência(s)