Evidence map›Paper›PMID 23442094›Full record

ReviewHuman gene therapy methods2013

Pre-existing anti-adeno-associated virus antibodies as a challenge in AAV gene therapy.

Vedell Louis Jeune, Jakob A Joergensen, Roger J Hajjar, Thomas Weber

Open access · greenAbstract readReview
In one paragraph

Review in Human gene therapy methods, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 221 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
221citing papers in PubMed, 1 pooled it
9.3field-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

221 citing papers in PubMed, 1 synthesis or guideline pooled it, 339 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Review
  4. Article
  5. Article
  6. Tissue-specific gene delivery approaches.Bioengineering & translational medicine · 2026
    Review
  7. Immune Toxicities in AAV Gene Therapy: Overview for Clinicians.International journal of molecular sciences · 2026
    Review
  8. Review
  9. Review
  10. Review
  11. Extracellular vesicles-associated AAVs for the treatment of Machado-Joseph disease.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Article
  12. Gene therapy for cardiac arrhythmias.Nature reviews. Cardiology · 2026
    Review
  13. Article
  14. Review
  15. Article
  16. Review
  17. The amazing AAV capsids: Into the structure-verse.Molecular therapy. Methods & clinical development · 2025
    Review
  18. Article
  19. Review
  20. Article

161 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

4 authors at 1 institution in 1 country.

Vedell Louis JeuneCardiovascular Research Center, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA.
Jakob A Joergensen
Roger J Hajjar
Thomas Weber
Icahn School of Medicine at Mount Sinai · US

Funding

TRAINING PROGRAM IN MOLECULAR AND CELLULAR CARDIOLOGYT32HL007824 · NHLBI · MOUNT SINAI SCHOOL OF MEDICINE OF NYU · PI GELB, BRUCE D · 1995 to 2022
$8.6M
C-TRIP: Targeted Gene Therapy for the Treatment of Heart Failure (P20)P20HL100396 · NHLBI · ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI · PI HAJJAR, ROGER J. · 2010 to 2011
$1.7M
Gene Therapy with Cardiotropic Vectors for the Treatment of Heart FailureR01HL088434 · NHLBI · ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI · PI HAJJAR, ROGER J. · 2010 to 2013
$1.7M
NHLBI NIH HHS HL007824NHLBI NIH HHS HL088434NHLBI NIH HHS HL100396NHLBI NIH HHS P20 HL100396NHLBI NIH HHS R01 HL088434NHLBI NIH HHS T32 HL007824
6 · The paper itself

Abstract

Adeno-associated virus (AAV)-based vectors are promising tools for gene therapeutic applications, in part because AAVs are nonpathogenic viruses, and vectors derived from them can drive long-term transgene expression without integration of the vector DNA into the host genome. AAVs are not strongly immunogenic, but they can, nonetheless, give rise to both a cellular and humoral immune response. As a result, a significant fraction of potential patients for AAV-based gene therapy harbors pre-existing antibodies against AAV. Because even very low levels of antibodies can prevent successful transduction, antecedent anti-AAV antibodies pose a serious obstacle to the universal application of AAV gene therapy. In this review, we discuss the current knowledge of the role of anti-AAV antibodies in AAV-based gene therapy with a particular emphasis on approaches to overcome the hurdle that they pose.

Indexed as

Genetic TherapyAnimalsAntibodies, NeutralizingAntibodies, ViralCapsid ProteinsDependovirusGenetic VectorsHumansImmunity, CellularImmunity, HumoralMutationPlasmapheresisSeroepidemiologic StudiesTransduction, GeneticAntibodies, NeutralizingAntibodies, ViralCapsid Proteins

Identifiers

PMID23442094
PMCPMC3732124
OpenAlexW2053820810

What OpenQuestion holds

Textmetadata
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.