Evidence map›Paper›PMID 41167625›Full record

ArticleHuman gene therapy2026

Enhancing AAV9-UFμDys1 Gene Therapy Efficacy Through Immunosuppression in Mice with Pre-Existing Immunity and Enabling Redosing Strategies for Duchenne Muscular Dystrophy.

Madhurima Saha, Radhika Bhake, Craig A Meyers, Kirsten Coleman, Prasad D Trivedi, Mitch Gallman, Moanaro Biswas, Roland W Herzog, Barry J Byrne, Manuela Corti

Erratum issuedAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Human gene therapy, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Article
  2. Review
  3. Article
4 · The record

Corrections and comments

  • Erratum issued
    2026
5 · Who and what money

Authors and funding

10 authors.

Madhurima SahaDepartment of Pediatrics, College of Medicine, University of Florida, Gainesville, Florida, USA.
Radhika BhakeDepartment of Pediatrics, College of Medicine, University of Florida, Gainesville, Florida, USA.
Craig A MeyersDepartment of Pediatrics, College of Medicine, University of Florida, Gainesville, Florida, USA.
Kirsten ColemanDepartment of Pediatrics, College of Medicine, University of Florida, Gainesville, Florida, USA.
Prasad D TrivediDepartment of Pediatrics, College of Medicine, University of Florida, Gainesville, Florida, USA.
Mitch GallmanDepartment of Pediatrics, College of Medicine, University of Florida, Gainesville, Florida, USA.
Moanaro BiswasDepartment of Pediatrics, School of Medicine, Indiana University, Indianapolis, Indiana, USA.
Roland W HerzogDepartment of Pediatrics, School of Medicine, Indiana University, Indianapolis, Indiana, USA.
Barry J ByrneDepartment of Pediatrics, College of Medicine, University of Florida, Gainesville, Florida, USA.
Manuela CortiDepartment of Pediatrics, College of Medicine, University of Florida, Gainesville, Florida, USA.

Funding

Immunomodulation Approaches to Improve Safety And Efficacy of Gene Therapy Treatment in Friedreich’s AtaxiaU01NS116752 · NINDS · UNIVERSITY OF FLORIDA · PI CORTI, MANUELA · 2021 to 2025
$3.8M
NINDS NIH HHS U01 NS116752
6 · The paper itself

Abstract

Significant progress has been made in gene therapy for Duchenne muscular dystrophy (DMD), a severe genetic disorder primarily affecting pediatric patients. However, the immune responses triggered by high-dose systemic delivery of adeno-associated virus (AAV) vectors remain a major challenge. These responses include the generation of long-lasting anti-capsid antibodies and potential immunity against the therapeutic transgene, rendering gene therapy ineffective. In addition, pre-existing anti-AAV antibodies exclude patients from eligibility for treatment. To address these limitations, we have developed an immunosuppression (IMS) strategy aimed at mitigating immune responses to the AAV capsid while enhancing microdystrophin expression. Using an optimized expression cassette (AAV9-UFµDys1) for sustained microdystrophin expression in striated muscle and heart, we observed a 40% improvement in muscle force compared with animals receiving a GFP-encoding control AAV9 vector. In

Indexed as

DependovirusDystrophinGenetic TherapyGenetic VectorsImmunosuppression TherapyMuscular Dystrophy, DuchenneAnimalsDisease Models, AnimalHumansMiceMice, Inbred mdxMuscle, SkeletalTransgenesDystrophinAAV gene therapyDMDdystrophinimmune responsepreexisting antibodiesredosing AAV

Identifiers

PMID41167625
PMCPMC13548098

What OpenQuestion holds

Textmetadata
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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.