| Popis: |
Adeno-associated virus (AAV) is now one of the top gene delivery/ gene therapy vectors in use today [1-6]. The hallmark goal of gene therapy is to deliver a therapeutic gene which then counteracts a negative phenotype or disease within the patient or animal model. While there are many types of diseases to treat, each disease is somewhat different and there are a variety of delivery/expression strategies which can be undertaken. AAV capsid type and tropism clearly play a valuable role in gene therapy; however the strategy for expressing the transgene is even more important. Here we discuss the three most prominent types of gene expression approaches, that is, which transcriptional promoter should be chosen for expressing the therapeutic gene. The issue is that many, perhaps all, therapeutic genes will likely have consequences, adverse reactions, if expressed at high levels. Yet the gene therapy agents must be safe, and not induce widespread unintended damage. |
