Evidence map›Paper›PMID 41520173›Full record

SynthesisMolecular therapy : the journal of the American Society of Gene Therapy2026

Incidence, timing, and clinical significance of adverse immune events after gene replacement therapy: A systematic review and meta-analysis.

Niccolò Maurizi, Enrico Ammirati, Elizabeth Silver, Kimberly Hong, Quan Bui, Alessia Argirò, Iacopo Olivotto, Eric D Adler

Abstract readSystematic ReviewMeta-Analysis
In one paragraph

Synthesis in Molecular therapy : the journal of the American Society of Gene Therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing 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

4 citing papers in PubMed.

  1. Article
  2. Review
  3. Review
  4. 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.

Niccolò MauriziCardiomyopathy Unit, Careggi University Hospital, Florence, Italy; Service of Cardiology, University Hospital of Lausanne (CHUV) and University of Lausanne (Unil), Lausanne, Switzerland. Electronic address: niccolo.maurizi@chuv.ch.
Enrico AmmiratiDe Gasperis Cardio Center, Transplant Center, Niguarda Hospital, Milano, Italy.
Elizabeth SilverDivision of Cardiovascular Medicine, Department of Medicine, University of California, San Diego, San Diego, CA, USA.
Kimberly HongDivision of Cardiovascular Medicine, Department of Medicine, University of California, San Diego, San Diego, CA, USA.
Quan BuiDivision of Cardiovascular Medicine, Department of Medicine, University of California, San Diego, San Diego, CA, USA.
Alessia ArgiròCardiomyopathy Unit, Careggi University Hospital, Florence, Italy.
Iacopo OlivottoCardiomyopathy Unit, Careggi University Hospital, Florence, Italy; Cardiology Unit, IRCCS Meyer's Children Hospital, Florence, Italy.
Eric D AdlerDivision of Cardiovascular Medicine, Department of Medicine, University of California, San Diego, San Diego, CA, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Adeno-associated virus (AAV)-based gene replacement has emerged as a transformative platform for severe genetic disorders, yet immune-mediated adverse events (AEs) pose significant barriers to widespread clinical adoption. We performed a systematic review and meta-analysis of prospective and retrospective studies of AAV gene therapy published between January 2005 and March 2025 (PROSPERO CRD420251046546). Data from 801 studies encompassing 1,972 patients and 2,142 patient-years were pooled to estimate the incidence and clinical impact of immunotoxicity. Random-effects meta-analysis yielded a 30.0% (95% CI, 22.5-38.9; I

Indexed as

DependovirusGenetic TherapyGenetic VectorsGene Therapy AgentsHumansIncidenceAAV therapyadverse eventsgene replacement

Identifiers

PMID41520173
PMCPMC12974193

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.