Evidence map›Paper›PMID 41282494›Full record

ArticleRegenerative therapy2025

Systemic MyoAAV-saRNA delivery activates endogenous utrophin and rescues dystrophic pathology in mdx mice.

Zhikai Xiahou, Tianyuan Wang, Zilian Zhang, Na Li, Hong Xiao, Zhuo Li, Yanming Gan, Xinwei Hou, Yafeng Song

Abstract readLetter
PubMed Full text at PMC Publisher
In one paragraph

Article in Regenerative therapy, 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

9 authors.

Zhikai XiahouChina Institute of Sport and Health Science, Beijing Sport University, No. 48, Xinxi Road, Haidian District, Beijing, China.
Tianyuan WangSchool of Sports Science, Beijing Sport University, No. 48, Xinxi Road, Haidian District, Beijing, China.
Zilian ZhangSchool of Sports Science, Beijing Sport University, No. 48, Xinxi Road, Haidian District, Beijing, China.
Na LiDepartment of Aerospace Medical Training, School of Aerospace Medicine, Air Force Medical University, No. 169, Changle West Road, Xi'an, China.
Hong XiaoPhysical Education Department, Northeastern University, No. 11, Lane 3, Wenhua Road, Heping District, Shenyang, China.
Zhuo LiSchool of Sports Science, Beijing Sport University, No. 48, Xinxi Road, Haidian District, Beijing, China.
Yanming GanSchool of Sports Science, Beijing Sport University, No. 48, Xinxi Road, Haidian District, Beijing, China.
Xinwei HouSchool of Physical Education, Hainan Normal University, No. 99, Longkun South Road, Haikou, China.
Yafeng SongChina Institute of Sport and Health Science, Beijing Sport University, No. 48, Xinxi Road, Haidian District, Beijing, China.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: Therapeutic activation of endogenous utrophin, a homolog of dystrophin, represents a mutation-agnostic therapeutic strategy with considerable potential for treating Duchenne muscular dystrophy (DMD). However, the large size of current dCas9-based activation systems hinders efficient adeno-associated virus (AAV) delivery, and the long-term feasibility and therapeutic durability of such treatments remain uncertain. To overcome these limitations, we developed a muscle-targeted utrophin activation platform based on small activating RNA (saRNA), termed MyoAAV-saRNA. Methods: Screening of a panel of saRNAs targeting the Results: Following intravenous delivery of MyoAAV 2A-saRNA-257 in mdx mouse models, significant upregulation of utrophin mRNA was observed. This activation led to functional improvements, including partial restoration of the sarcolemma, histopathological amelioration (reduced centronucleation, inflammation, and fibrosis), and enhanced muscle strength. Conclusion: Collectively, these preclinical findings establish MyoAAV-saRNA as a promising and translatable platform for utrophin induction, highlighting its therapeutic potential for DMD.

Indexed as

Duchenne muscular dystrophyGene therapyMyoAAVsaRNAUtrophin

Identifiers

PMID41282494
PMCPMC12636391

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.