Evidence map›Paper›PMID 41183732›Full record

ArticleKidney international2026

Mouse Alport podocytes are susceptible to AAV9 transduction in vivo.

Meei-Hua Lin, Kohei Omachi, Joshua F Begin, Jennifer L Richardson, Jeffrey H Miner

Abstract read
In one paragraph

Article in Kidney international, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

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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

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3 · Its place in the literature

Who cites it

2 citing papers in PubMed.

  1. Article
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4 · The record

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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

5 authors.

Meei-Hua LinDivision of Nephrology, Department of Medicine, Washington University School of Medicine, St. Louis, Missouri, USA.
Kohei OmachiDivision of Nephrology, Department of Medicine, Washington University School of Medicine, St. Louis, Missouri, USA; Laboratory for Tissue Microenvironment, RIKEN Center for Biosystems Dynamics Research (BDR), Kobe, Japan.
Joshua F BeginDivision of Nephrology, Department of Medicine, Washington University School of Medicine, St. Louis, Missouri, USA.
Jennifer L RichardsonDivision of Nephrology, Department of Medicine, Washington University School of Medicine, St. Louis, Missouri, USA.
Jeffrey H MinerDivision of Nephrology, Department of Medicine, Washington University School of Medicine, St. Louis, Missouri, USA. Electronic address: jeffminer@wustl.edu.

Funding

Resource Based Center for Musculoskeletal Biology and Medicine (Overall Application)P30AR074992 · NIAMS · WASHINGTON UNIVERSITY · PI MATTHEW J SILVA · 2019 to 2026
$6.8M
Innovative Approaches to Treating Alport SyndromeR01DK128660 · NIDDK · WASHINGTON UNIVERSITY · PI MINER, JEFFREY H · 2021 to 2025
$2.2M
NIAMS NIH HHS P30 AR074992NIDDK NIH HHS R01 DK128660
6 · The paper itself

Abstract

introductionAlport syndrome features a defective glomerular basement membrane (GBM) due to variants in COL4A3, COL4A4, and COL4A5. The most severe forms, which lack the GBM's collagen α3α4α5(IV) network, progress from hematuria in early childhood to proteinuria, chronic kidney disease, and kidney failure by the age of ∼30. As a monogenic disease without specific treatments, and with podocytes being the only glomerular cells that synthesize collagen α3α4α5(IV), the ability to efficiently deliver genes to Alport podocytes could open up new possibilities for treatment.

methodsAs a proof-of-concept study, we investigated whether podocytes in X-linked Alport mice were susceptible to transduction by adeno-associated virus (AAV)9. ssAAV9-CAG-tdTomato and scAAV9-CMV-Cre were intravenously injected into Col4a5

resultsssAAV9-CAG-tdTomato delivered to controls failed to transduce podocytes, but heterozygous female and hemizygous male Alport podocytes showed a range of transduction efficiencies, from 1.8% to 26%, which correlated positively with levels of albuminuria. Similar correlation between podocyte transduction and albuminuria was observed in the more sensitive Ai14 system with scAAV9-CMV-Cre administration. A subset of tubular and mesangial cells could also be transduced, the former in Alport mice and the latter in both Alport and control mice.

conclusionsThe Alport GBM becomes leaky to AAV9 as mice mature, allowing viruses to reach and transduce a substantial subset of podocytes. This is promising for someday using AAV9 or other vehicles in gene therapy for patients with Alport syndrome. Interestingly, mesangial cells of control and young Alport mice were moderately susceptible to transduction, demonstrating that gene delivery to mesangial cells in mice is a viable approach for investigating mesangial cell biology in any context.

Indexed as

DependovirusGenetic VectorsNephritis, HereditaryPodocytesTransduction, GeneticAnimalsCollagen Type IVDisease Models, AnimalFemaleGenetic TherapyGlomerular Basement MembraneHumansMaleMiceMicrofilament ProteinsCol4a5 protein, mouseCollagen Type IVMicrofilament ProteinsSynpo protein, mouseAAV9Alport syndromeglomerular basement membranepodocyte

Identifiers

PMID41183732
PMCPMC12831642

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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.