Targeted introduction and effective expression of hFIX at the AAVS1 locus in mesenchymal stem cells.
Li, Shu-Jun; Luo, Ying; Zhang, Le-Meng; Yang, Wei; Zhang, Guo-Gang.
Mol Med Rep
; 15(3): 1313-1318, 2017 Mar.
Artigo em Inglês | MEDLINE | ID: mdl-28112377
Ribosomal DNA integrating rAAV-rDNA vectors allow for stable transgene expression.
Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques.
A 16-bp RBE element mediated Rep-dependent site-specific integration in AAVS1 transgenic mice for expression of hFIX.
AAV vectors containing rDNA homology display increased chromosomal integration and transgene persistence.
Significant differences in integration sites of Moloney murine leukemia virus/Moloney murine sarcoma virus retroviral vector carrying recombinant coagulation factor IX in two human cell lines.
High-throughput sequencing reveals principles of adeno-associated virus serotype 2 integration.
Adeno-associated virus type 2 preferentially integrates single genome copies with defined breakpoints.
Sites of retroviral DNA integration: From basic research to clinical applications.
Genome Engineering Using Adeno-associated Virus: Basic and Clinical Research Applications.
Promoterless gene targeting without nucleases ameliorates haemophilia B in mice.