Evidence map›Paper›PMID 37291262›Full record

ArticleNature methods2023

Hardwiring tissue-specific AAV transduction in mice through engineered receptor expression.

James Zengel, Yu Xin Wang, Jai Woong Seo, Ke Ning, James N Hamilton, Bo Wu, Marina Raie, Colin Holbrook, Shiqi Su, Derek R Clements and 10 more

Open access · hybridAbstract read
In one paragraph

Article in Nature methods, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 papers.

0numbers the graph read from it
0cells of the map it votes in
31citing papers in PubMed
9.4field-weighted citation impact, top 2% of its field
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

31 citing papers in PubMed, 31 citations in OpenAlex.

  1. Article
  2. Article
  3. Article
  4. Review
  5. Review
  6. Review
  7. Article
  8. Article
  9. Epigenome editing based treatment: Progresses and challenges.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Review
  10. Article
  11. Article
  12. Article
  13. Article
  14. Review
  15. Review
  16. AAVR Expression is Essential for AAV Vector Transduction in Sensory Hair Cells.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025
    Article
  17. Review
  18. A synthetic opsin restores vision in patients with severe retinal degeneration.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
  19. Review
  20. Article
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

20 authors at 4 institutions in 1 country.

James Zengel *Department of Microbiology and Immunology, Stanford University School of Medicine, Stanford, CA, USA.
Yu Xin Wang *Baxter Laboratory for Stem Cell Biology, Department of Microbiology and Immunology, Stanford University School of Medicine, Stanford, CA, USA.
Jai Woong Seo *Department of Radiology, Stanford University School of Medicine, Stanford, CA, USA.ORCID http://orcid.org/0000-0002-2732-7498
Ke Ning *Department of Ophthalmology, Stanford University School of Medicine, Stanford, CA, USA.
James N HamiltonBaxter Laboratory for Stem Cell Biology, Department of Microbiology and Immunology, Stanford University School of Medicine, Stanford, CA, USA.
Bo WuDepartment of Radiology, Stanford University School of Medicine, Stanford, CA, USA.
Marina RaieDepartment of Radiology, Stanford University School of Medicine, Stanford, CA, USA.ORCID http://orcid.org/0000-0002-5549-404X
Colin HolbrookBaxter Laboratory for Stem Cell Biology, Department of Microbiology and Immunology, Stanford University School of Medicine, Stanford, CA, USA.
Shiqi SuBaxter Laboratory for Stem Cell Biology, Department of Microbiology and Immunology, Stanford University School of Medicine, Stanford, CA, USA.
Derek R ClementsDepartment of Microbiology and Immunology, Stanford University School of Medicine, Stanford, CA, USA.
Sirika PillayDepartment of Microbiology and Immunology, Stanford University School of Medicine, Stanford, CA, USA.
Andreas S PuschnikDepartment of Microbiology and Immunology, Stanford University School of Medicine, Stanford, CA, USA.ORCID http://orcid.org/0000-0002-9605-9458
Monte M WinslowDepartment of Genetics, Stanford University School of Medicine, Stanford, CA, USA.ORCID http://orcid.org/0000-0002-5730-9573
Juliana IdoyagaDepartment of Microbiology and Immunology, Stanford University School of Medicine, Stanford, CA, USA.ORCID http://orcid.org/0000-0002-4430-8862
Claude M NagamineDepartment of Comparative Medicine, Stanford University School of Medicine, Stanford, CA, USA.
Yang SunDepartment of Ophthalmology, Stanford University School of Medicine, Stanford, CA, USA.ORCID http://orcid.org/0000-0001-8735-4765
Vinit B MahajanDepartment of Ophthalmology, Stanford University School of Medicine, Stanford, CA, USA.
Katherine W FerraraDepartment of Radiology, Stanford University School of Medicine, Stanford, CA, USA.ORCID http://orcid.org/0000-0002-4976-9107
Helen M BlauBaxter Laboratory for Stem Cell Biology, Department of Microbiology and Immunology, Stanford University School of Medicine, Stanford, CA, USA.ORCID http://orcid.org/0000-0001-6503-5480
Jan E CaretteDepartment of Microbiology and Immunology, Stanford University School of Medicine, Stanford, CA, USA. carette@stanford.edu.ORCID http://orcid.org/0000-0002-5187-8070
Stanford University · USPalo Alto Veterans Institute for Research · USDiscovery Institute · USStanford Medicine · US

Funding

MOLECULAR BASIS OF HOST-PARASITE INTERACTIONT32AI007328 · NIAID · STANFORD UNIVERSITY · PI JUSTIN L SONNENBURG · 1987 to 2026
$11.9M
Stanford Vision Research CoreP30EY026877 · NEI · STANFORD UNIVERSITY · PI Alfredo Dubra · 2017 to 2026
$8.0M
Stem Cells for Brain and BrawnR01AG020961 · NIA · STANFORD UNIVERSITY · PI BLAU, HELEN M · 2002 to 2019
$5.0M
In vivo PET imaging of novel engineered AAVs informs capsid design in Alzheimer's DiseaseR01EB028646 · NIBIB · STANFORD UNIVERSITY · PI FERRARA, KATHERINE W · 2019 to 2022
$3.8M
Human 3D neuro-muscular assembloids to study cell tropism and host factor utilization of divergent neuropathogenic enterovirusesR01AI169467 · NIAID · STANFORD UNIVERSITY · PI Jan E Carette, Sergiu Pasca · 2022 to 2026
$3.7M
The role of primary cilia in glaucoma pathogenesisR01EY025295 · NEI · STANFORD UNIVERSITY · PI Yang Sun · 2016 to 2026
$3.5M
Transitional dendritic cells: identifying the origin and role of a novel innate immune population during viral infectionR01AI158808 · NIAID · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI Juliana Idoyaga · 2021 to 2026
$3.5M
Host determinants of enterovirus RNA replication and in vivo neuropathogenesisR01AI153169 · NIAID · STANFORD UNIVERSITY · PI CARETTE, JAN E · 2021 to 2025
$2.7M
Regulation of eicosanoid signaling lipids to improve skeletal muscle function and increase healthspan during agingR01AG069858 · NIA · STANFORD UNIVERSITY · PI BLAU, HELEN M · 2020 to 2023
$2.4M
Genetic screens to find critical host factors for SARS-CoV-2 infectionR01AI140186 · NIAID · STANFORD UNIVERSITY · PI CARETTE, JAN E · 2019 to 2023
$2.3M
Inflammatory Gene Transcription in the RetinaR01EY030151 · NEI · STANFORD UNIVERSITY · PI BASSUK, ALEXANDER G, MAHAJAN, VINIT B · 2020 to 2024
$2.0M
Host determinants of adeno-associated virus entry and traffickingR01AI130123 · NIAID · STANFORD UNIVERSITY · PI CARETTE, JAN E · 2018 to 2022
$2.0M
CSRD VA I01 CX001481NCI NIH HHS R01 CA219994NEI NIH HHS P30 EY026877NEI NIH HHS R01 EY024665NEI NIH HHS R01 EY025225NEI NIH HHS R01 EY025295NEI NIH HHS R01 EY030151NEI NIH HHS R01 EY032159NIAID NIH HHS R01 AI130123NIAID NIH HHS R01 AI140186NIAID NIH HHS R01 AI141970NIAID NIH HHS R01 AI153169NIAID NIH HHS R01 AI158808NIAID NIH HHS R01 AI169467NIAID NIH HHS T32 AI007328NIA NIH HHS R01 AG020961NIA NIH HHS R01 AG069858NIBIB NIH HHS R01 EB028646NINDS NIH HHS K99 NS120278NINDS NIH HHS R00 NS120278
6 · The paper itself

Abstract

The development of transgenic mouse models that express genes of interest in specific cell types has transformed our understanding of basic biology and disease. However, generating these models is time- and resource-intensive. Here we describe a model system, SELective Expression and Controlled Transduction In Vivo (SELECTIV), that enables efficient and specific expression of transgenes by coupling adeno-associated virus (AAV) vectors with Cre-inducible overexpression of the multi-serotype AAV receptor, AAVR. We demonstrate that transgenic AAVR overexpression greatly increases the efficiency of transduction of many diverse cell types, including muscle stem cells, which are normally refractory to AAV transduction. Superior specificity is achieved by combining Cre-mediated AAVR overexpression with whole-body knockout of endogenous Aavr, which is demonstrated in heart cardiomyocytes, liver hepatocytes and cholinergic neurons. The enhanced efficacy and exquisite specificity of SELECTIV has broad utility in development of new mouse model systems and expands the use of AAV for gene delivery in vivo.

Indexed as

Genetic VectorsGene Transfer TechniquesAnimalsDependovirusGenetic TherapyMiceMice, TransgenicTransduction, GeneticTransgenes

Identifiers

PMID37291262
PMCPMC10333121
OpenAlexW4379878663

What OpenQuestion holds

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LicenceCC BY
Read underepoch 390

Registered trials

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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.