| Products/Services Used | Details | Operation |
|---|---|---|
| Codon Optimization | To produce AAV constructs containing alternative sequences between the left-hand ITR and the eGFP start codon, a 5′ AAV ITR sequence was synthesized (Genscript) with a downstream insert of 105 random nucleotides (R105) flanked by NotI and XbaI recognition sites. The ITR-R105 sequence was subcloned into p∆LSP1-eGFP to replace the 5′ ITR (up to the Kozac consensus sequence of the eGFP start codon) to produce pAAV-insert-eGFP. DNA sequences (synthesized by Genscript) were subcloned into this construct after NotI and XbaI restriction digestion to produce vectors carrying different sequences between the ITR and the eGFP start codon. | Get A Quote |
Vectors based on adeno-associated virus type 2 (AAV2) are powerful tools for gene transfer and genome editing applications. The level of interest in this system has recently surged in response to reports of therapeutic efficacy in human clinical trials， most notably for those in patients with hemophilia B (ref. 3). Understandably， a recent report drawing an association between AAV2 integration events and human hepatocellular carcinoma (HCC) has generated controversy about the causal or incidental nature of this association and the implications for AAV vector safety. Here we describe and functionally characterize a previously unknown liver-specific enhancer-promoter element in the wild-type AAV2 genome that ... More
