[HTML][HTML] Adeno-associated virus-mediated gene transfer to renal tubule cells via a retrograde ureteral approach
DC Chung, B Fogelgren, KM Park, J Heidenberg… - Nephron Extra, 2011 - karger.com
Nephron Extra, 2011•karger.com
Background/Aims: Gene therapy involves delivery of exogenous DNA to provide a
therapeutic protein. Ideally, a gene therapy vector should be non-toxic, non-immunogenic,
easy to produce, and efficient in protecting and delivering DNA into target cells. Methods:
Adeno-associated virus (AAV) offers these advantages and few, if any, disadvantages, and
over 100 isolates exist. We previously showed that AAV-mediated gene therapy can be used
to restore vision to patients with Leber's congenital amaurosis, a disease of childhood …
therapeutic protein. Ideally, a gene therapy vector should be non-toxic, non-immunogenic,
easy to produce, and efficient in protecting and delivering DNA into target cells. Methods:
Adeno-associated virus (AAV) offers these advantages and few, if any, disadvantages, and
over 100 isolates exist. We previously showed that AAV-mediated gene therapy can be used
to restore vision to patients with Leber's congenital amaurosis, a disease of childhood …
Background/Aims
Gene therapy involves delivery of exogenous DNA to provide a therapeutic protein. Ideally, a gene therapy vector should be non-toxic, non-immunogenic, easy to produce, and efficient in protecting and delivering DNA into target cells. Methods
Adeno-associated virus (AAV) offers these advantages and few, if any, disadvantages, and over 100 isolates exist. We previously showed that AAV-mediated gene therapy can be used to restore vision to patients with Leber’s congenital amaurosis, a disease of childhood blindness. Results
Here we show that novel recombinant AAV2/8 and AAV2/9 transduce kidney tubule cells with high efficiency both in vitroin cell culture and in vivoin mice. In addition, we adapted and modified a retrograde approach to allow for optimal transgene delivery to renal tubular cells that further minimizes the risk of an immunogenic reaction. Conclusions
We believe that recombinant AAV2, especially AAV2/8, gene delivery to renal tubule cells via a retrograde approach represents a viable method for gene therapy for a multitude of renal disorders ranging from autosomal dominant polycystic kidney disease to acute kidney injury.Karger