Evidence map›Paper›PMID 31836454›Full record

ReviewPharmacology & therapeutics2020

Adeno-associated viral vector-mediated immune responses: Understanding barriers to gene delivery.

Natalie F Nidetz, Michael C McGee, Longping V Tse, Chengwen Li, Le Cong, Yunxing Li, Weishan Huang

Open access · hybridAbstract readReview
In one paragraph

Review in Pharmacology & therapeutics, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 96 papers.

0numbers the graph read from it
0cells of the map it votes in
96citing papers in PubMed
14.5field-weighted citation impact, top 1% 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

96 citing papers in PubMed, 164 citations in OpenAlex.

  1. Article
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  8. A Translational Roadmap for Neurological Nonsense Mutation Disorders.International journal of molecular sciences · 2026
    Review
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  19. Article
  20. Targets and Gene Therapy of ALS (Part 1).International journal of molecular sciences · 2025
    Review

36 more citing papers are in PubMed but not listed here.

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

7 authors at 4 institutions in 1 country.

Natalie F NidetzDepartment of Pathobiological Sciences, School of Veterinary Medicine, Louisiana State University, Baton Rouge, LA 70803, USA.
Michael C McGeeDepartment of Pathobiological Sciences, School of Veterinary Medicine, Louisiana State University, Baton Rouge, LA 70803, USA.
Longping V TseDepartment of Epidemiology, School of Public Health, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Chengwen LiGene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Le CongDepartment of Pathology and Department of Genetics, School of Medicine, Stanford University, Stanford, CA 94305, USA.
Yunxing LiDepartment of Molecular Medicine, Cornell University, Ithaca, NY 14853, USA.
Weishan HuangDepartment of Pathobiological Sciences, School of Veterinary Medicine, Louisiana State University, Baton Rouge, LA 70803, USA; Department of Microbiology and Immunology, Cornell University, Ithaca, NY 14853, USA. Electronic address: huang1@lsu.edu.
Louisiana State University · USUniversity of North Carolina at Chapel Hill · USCornell University · USStanford University · US

Funding

The role of adipocyte-driven inflammation and leptin pathway in pulmonary viral infections caused by SARS-CoV-2 and influenza A virusesP20GM130555 · NIGMS · LOUISIANA STATE UNIV A&M COL BATON ROUGE · PI Mariano Carossino · 2019 to 2026
$19.9M
Protein CoreP30GM110760 · NIGMS · LOUISIANA STATE UNIV A&M COL BATON ROUGE · PI KOUSOULAS, KONSTANTIN G · 2014 to 2018
$5.6M
Optimization of AAV vector to deliver FVa for hemophilia with inhibitorsR01HL144661 · NHLBI · UNIV OF NORTH CAROLINA CHAPEL HILL · PI LI, CHENGWEN, NICHOLS, TIMOTHY CHARLES · 2019 to 2022
$2.2M
Enhance AAV Liver Transduction with Capsid Immune EvasionR01AI117408 · NIAID · UNIV OF NORTH CAROLINA CHAPEL HILL · PI LI, CHENGWEN, SAMULSKI, RICHARD J · 2016 to 2020
$2.0M
Directed evolution of AAV vectors for hemophilia to evade neutralizationR01HL125749 · NHLBI · UNIV OF NORTH CAROLINA CHAPEL HILL · PI LI, CHENGWEN, NICHOLS, TIMOTHY CHARLES · 2015 to 2018
$1.5M
A novel murine model of allergic airway inflammation.R21AI138497 · NIAID · CORNELL UNIVERSITY · PI AUGUST, AVERY, HUANG, WEISHAN · 2018 to 2019
$443k
Itk signaling and Type 1 regulatory T cell differentiation and function in allergic airway inflammationR21AI129422 · NIAID · CORNELL UNIVERSITY · PI AUGUST, AVERY, HUANG, WEISHAN · 2018 to 2019
$431k
GCN2 signaling in pathogenic and regulatory T cell development in allergic airway inflammationR21AI137822 · NIAID · LOUISIANA STATE UNIV A&M COL BATON ROUGE · PI HUANG, WEISHAN · 2018 to 2019
$407k
NHLBI NIH HHS R01 HL125749NHLBI NIH HHS R01 HL144661NIAID NIH HHS R01 AI117408NIAID NIH HHS R21 AI129422NIAID NIH HHS R21 AI137822NIAID NIH HHS R21 AI138497NIGMS NIH HHS P20 GM130555NIGMS NIH HHS P30 GM110760
6 · The paper itself

Abstract

Adeno-associated viral (AAV) vectors have emerged as the leading gene delivery platform for gene therapy and vaccination. Three AAV-based gene therapy drugs, Glybera, LUXTURNA, and ZOLGENSMA were approved between 2012 and 2019 by the European Medicines Agency and the United States Food and Drug Administration as treatments for genetic diseases hereditary lipoprotein lipase deficiency (LPLD), inherited retinal disease (IRD), and spinal muscular atrophy (SMA), respectively. Despite these therapeutic successes, clinical trials have demonstrated that host anti-viral immune responses can prevent the long-term gene expression of AAV vector-encoded genes. Therefore, it is critical that we understand the complex relationship between AAV vectors and the host immune response. This knowledge could allow for the rational design of optimized gene transfer vectors capable of either subverting host immune responses in the context of gene therapy applications, or stimulating desirable immune responses that generate protective immunity in vaccine applications to AAV vector-encoded antigens. This review provides an overview of our current understanding of the AAV-induced immune response and discusses potential strategies by which these responses can be manipulated to improve AAV vector-mediated gene transfer.

Indexed as

Gene Transfer TechniquesAntibodies, NeutralizingAntigen-Presenting CellsDependovirusGene EditingGenetic VectorsImmune ToleranceImmunity, InnateT-LymphocytesAntibodies, NeutralizingAdeno-associated virusCytotoxic lymphocytesGene therapyImmune tolerancePre-existing immunityVaccination

Identifiers

PMID31836454
PMCPMC6980784
OpenAlexW2995820186

What OpenQuestion holds

Textmetadata
LicenceTDM
Read underepoch 390

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.