AAV-based neonatal gene therapy for hemophilia A: long-term correction and avoidance of immune responses in mice
Hu C, Lipshutz GS. AAV-based neonatal gene therapy for hemophilia A: long-term correction and avoidance of immune responses in mice. Gene Ther. 2012 Dec;19(12):1166-76. doi: 10.1038/gt.2011.200. Epub 2012 Jan 12. PMID: 22241178; PMCID: PMC3432168.
