| Adeno-Associated Virus-Mediated Gene Transfer to Renal Tubule Cells via a Retrograde Ureteral Approach. | |
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MedLine Citation:
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PMID: 22470395 Owner: NLM Status: In-Data-Review |
Abstract/OtherAbstract:
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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. |
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Authors:
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Daniel C Chung; Ben Fogelgren; Kwon Moo Park; Jessica Heidenberg; Xiaofeng Zuo; Liwei Huang; Jean Bennett; Joshua H Lipschutz |
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Publication Detail:
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Type: Journal Article Date: 2011-11-23 |
Journal Detail:
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Title: Nephron extra Volume: 1 ISSN: 1664-5529 ISO Abbreviation: Nephron Extra Publication Date: 2011 Jan |
Date Detail:
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Created Date: 2012-04-03 Completed Date: - Revised Date: - |
Medline Journal Info:
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Nlm Unique ID: 101571879 Medline TA: Nephron Extra Country: Switzerland |
Other Details:
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Languages: eng Pagination: 217-23 Citation Subset: - |
Affiliation:
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Department of Ophthalmology, University of Pennsylvania, Philadelphia, Pa., USA. |
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From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine
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