Evidence map›Paper›PMID 41155416›Full record

ReviewInternational journal of molecular sciences2025

Navigating the Hurdles of Intra-Articular AAV Gene Therapy.

Wenjun Li, Owen Thornton, Susi Feng, Chengwen Li

Abstract readReview
In one paragraph

Review in International journal of molecular sciences, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing 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

1 citing paper in PubMed.

  1. 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

4 authors.

Wenjun LiGene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Owen ThorntonDepartment of Psychology and Neuroscience, University of North Carolina at Chapel Hill, Chapel Hill, NC 27599, USA.
Susi FengGene Therapy Center, 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.ORCID 0000-0003-4397-4561

Funding

Novel strategy to block Nabs for AAV gene deliveryR01AI168242 · NIAID · UNIV OF NORTH CAROLINA CHAPEL HILL · PI Chengwen Li, Timothy Charles Nichols · 2022 to 2026
$3.0M
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
Development of AAV vectors for CF therapyR01HL151348 · NHLBI · UNIV OF NORTH CAROLINA CHAPEL HILL · PI LI, CHENGWEN, SAMULSKI, RICHARD J · 2021 to 2024
$1.6M
NHLBI NIH HHS R01 HL144661NHLBI NIH HHS R01 HL151348NIAID NIH HHS R01 AI168242NIH HHS 1R01HL144661-26
6 · The paper itself

Abstract

Joint diseases represent a significant health burden due to their high prevalence and morbidity, yet current treatments fail to provide comprehensive and long-term relief for all patients. In this context, adeno-associated virus (AAV) gene therapy has emerged as a promising approach, offering advantages such as prolonged efficacy and minimal immunogenicity. AAV has been extensively studied for various medical conditions, with some applications successfully implemented in patient treatments. Currently, a few clinical trials utilizing AAV have been completed for treating arthritis. However, challenges such as transduction efficiency, off-targets, and preexisting immune responses persist. This review provides an overview of the current paradigms of treatment with regard to joint diseases, elaborates on the AAV delivery barriers related to application in treating joint diseases, and discusses strategies to improve gene therapy efficacy, including AAV capsid engineering, small molecule-assisted AAV delivery, optimizing tissue-specific or inflammation-inducible promoters, as well as strategies to mitigate immune responses to AAV.

Indexed as

DependovirusGenetic TherapyGenetic VectorsJoint DiseasesAnimalsGene Transfer TechniquesHumansAAV engineeringadeno-associated virusbarriersimmune managementjoint

Identifiers

PMID41155416
PMCPMC12564301

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.