In:
Nephron Extra, S. Karger AG, Vol. 1, No. 1 ( 2011-11-23), p. 217-223
Abstract:
〈 i 〉 Background/Aims: 〈 /i 〉 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. 〈 i 〉 Methods: 〈 /i 〉 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. 〈 i 〉 Results: 〈 /i 〉 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. 〈 i 〉 Conclusions: 〈 /i 〉 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.
Type of Medium:
Online Resource
ISSN:
1664-5529
Language:
English
Publisher:
S. Karger AG
Publication Date:
2011
detail.hit.zdb_id:
2658188-7
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