Life-long correction of hyperbilirubinemia with a neonatal liver-specific AAV-mediated gene transfer in a lethal mouse model of Crigler-Najjar Syndrome.
Bortolussi G, Zentillin L, Vaníkova J, Bockor L, Bellarosa C, Mancarella A, Vianello E, Tiribelli C, Giacca M, Vitek L, Muro AF.
Bortolussi G, et al. Among authors: tiribelli c.
Hum Gene Ther. 2014 Sep;25(9):844-55. doi: 10.1089/hum.2013.233.
Hum Gene Ther. 2014.
PMID: 25072305
Free PMC article.
Taken together, our data support the concept that liver is the best organ for efficient and long-term CNSI gene therapy, and suggest that the use of extra-hepatic tissues should be coupled to the presence of bilirubin transporters....
Taken together, our data support the concept that liver is the best organ for efficient and long-term CNSI gene therapy, and suggest …