Evidence map›Paper›PMID 22470395›Full record

ArticleNephron extra2011

Adeno-Associated Virus-Mediated Gene Transfer to Renal Tubule Cells via a Retrograde Ureteral Approach.

Daniel C Chung, Ben Fogelgren, Kwon Moo Park, Jessica Heidenberg, Xiaofeng Zuo, Liwei Huang, Jean Bennett, Joshua H Lipschutz

Abstract read
In one paragraph

Article in Nephron extra, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 25 papers.

0numbers the graph read from it
0cells of the map it votes in
25citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

25 citing papers in PubMed.

  1. Trial
  2. Article
  3. Article
  4. Article
  5. Article
  6. Article
  7. Article
  8. Article
  9. Targeted therapeutic strategies for the kidney.Expert opinion on therapeutic targets · 2024
    Review
  10. In Vivo Assessment of Laboratory-Grown Kidney Tissue Grafts.Bioengineering (Basel, Switzerland) · 2023
    Article
  11. Cell and gene therapy for kidney disease.Nature reviews. Nephrology · 2023
    Review
  12. Article
  13. Article
  14. Article
  15. Review
  16. Improving Molecular Therapy in the Kidney.Molecular diagnosis & therapy · 2020
    Review
  17. Article
  18. Article
  19. Article
  20. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors.

Daniel C ChungDepartment of Ophthalmology, University of Pennsylvania, Philadelphia, Pa., USA.
Ben Fogelgren
Kwon Moo Park
Jessica Heidenberg
Xiaofeng Zuo
Liwei Huang
Jean Bennett
Joshua H Lipschutz

Funding

TRANSGENIC AND CHIMERIC MOUSE COREP30DK050306 · NIDDK · UNIVERSITY OF PENNSYLVANIA · PI REBECCA G WELLS · 1997 to 2026
$32.5M
The Exocyst in Synthesis, Cystogenesis and TubulogenesisR01DK069909 · NIDDK · UNIVERSITY OF PENNSYLVANIA · PI LIPSCHUTZ, JOSHUA H · 2005 to 2009
$1.3M
non-viral medicated gene targeting to the retinaK08EY017024 · NEI · UNIVERSITY OF PENNSYLVANIA · PI CHUNG, DANIEL · 2006 to 2010
$838k
The exocyst in kidney development and cyst formationK01DK087852 · NIDDK · UNIVERSITY OF PENNSYLVANIA · PI FOGELGREN, BENJAMIN C. · 2010 to 2014
$764k
NEI NIH HHS K08 EY017024NIDDK NIH HHS K01 DK087852NIDDK NIH HHS P30 DK050306NIDDK NIH HHS R01 DK069909
6 · The paper itself

Abstract

BACKGROUND/

aimsGene 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.

methodsAdeno-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.

resultsHere 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.

conclusionsWe 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.

Indexed as

Adeno-associated virusGene therapyKidney injury

Identifiers

PMID22470395
PMCPMC3290852

What OpenQuestion holds

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LicenceCC BY-NC-ND
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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.