Evidence map›Paper›PMID 41793215›Full record

ArticleJournal of cellular and molecular medicine2026

Bioengineered AAV9 and Optimised Microdystrophin Vectors Augment Phenotypic Rescue in a Murine Model of Duchenne Muscular Dystrophy.

Mohankumar B Senthilkumar, Sanya Sharma, Navaneeth Srinivasan, Anila Varghese, Pratiksha Sarangi, Vijayata Singh, Narendra Kumar, Devyani Yenurkar, Sudip Mukherjee, Sonal Amit and 5 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Journal of cellular and molecular medicine, 2026. 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. AAVcc47-The Yale journal of biology and medicine · 2026
    Article
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

15 authors.

Mohankumar B SenthilkumarDepartment of Biological Sciences and Bioengineering and Mehta Family Center for Engineering in Medicine and Gangwal School of Medical Sciences and Technology, Indian Institute of Technology, Kanpur, Uttar Pradesh, India.
Sanya SharmaDepartment of Biological Sciences and Bioengineering and Mehta Family Center for Engineering in Medicine and Gangwal School of Medical Sciences and Technology, Indian Institute of Technology, Kanpur, Uttar Pradesh, India.
Navaneeth SrinivasanDepartment of Biological Sciences and Bioengineering and Mehta Family Center for Engineering in Medicine and Gangwal School of Medical Sciences and Technology, Indian Institute of Technology, Kanpur, Uttar Pradesh, India.
Anila VargheseDepartment of Biological Sciences and Bioengineering and Mehta Family Center for Engineering in Medicine and Gangwal School of Medical Sciences and Technology, Indian Institute of Technology, Kanpur, Uttar Pradesh, India.
Pratiksha SarangiDepartment of Biological Sciences and Bioengineering and Mehta Family Center for Engineering in Medicine and Gangwal School of Medical Sciences and Technology, Indian Institute of Technology, Kanpur, Uttar Pradesh, India.
Vijayata SinghDepartment of Biological Sciences and Bioengineering and Mehta Family Center for Engineering in Medicine and Gangwal School of Medical Sciences and Technology, Indian Institute of Technology, Kanpur, Uttar Pradesh, India.
Narendra KumarDepartment of Biological Sciences and Bioengineering and Mehta Family Center for Engineering in Medicine and Gangwal School of Medical Sciences and Technology, Indian Institute of Technology, Kanpur, Uttar Pradesh, India.
Devyani YenurkarSchool of Biomedical Engineering, IIT (BHU), Varanasi, Uttar Pradesh, India.
Sudip MukherjeeSchool of Biomedical Engineering, IIT (BHU), Varanasi, Uttar Pradesh, India.ORCID https://orcid.org/0000-0002-3625-2272
Sonal AmitAutonomous State Medical College, Kanpur, Uttar Pradesh, India.
Sameer BhatiaInstitute of Medical Genetics and Genomics, Sir Gangaram Hospital, New Delhi, India.ORCID https://orcid.org/0000-0002-4997-3722
Santosh K MisraInstitute of Medical Genetics and Genomics, Sir Gangaram Hospital, New Delhi, India.
Ratna Dua PuriInstitute of Medical Genetics and Genomics, Sir Gangaram Hospital, New Delhi, India.
Jeffrey ChamberlainDepartment of Neurology, University of Washington School of Medicine, Seattle, Washington, USA.
Giridhara R JayandharanDepartment of Biological Sciences and Bioengineering and Mehta Family Center for Engineering in Medicine and Gangwal School of Medical Sciences and Technology, Indian Institute of Technology, Kanpur, Uttar Pradesh, India.

Funding

Indian Institute of Technology Kanpur intramural fundsThe Wellcome Trust DBT India Alliance IA/TSG/22/1/600401The Wellcome Trust DBT India Alliance Team Science grant
6 · The paper itself

Abstract

Duchenne muscular dystrophy (DMD) is a severe neuromuscular disorder without an effective cure. Adeno-associated virus (AAV) based gene therapy has improved dystrophin function, with sub-optimal clinical outcomes. We reasoned that a combination of rational engineering of AAV9 capsids modified at the post-translational modification sites, optimal promoter selection, and codon-optimisation of the microdystrophin (μDys) can enhance the AAV9 vector functionality. Our initial promoter screening demonstrated improved dystrophin expression in muscle fibres with a ubiquitous CAG promoter (1.61-fold in CAG vs. MHCK7, p < 0.0001) in mdx mice. We then evaluated two engineered AAV9 capsids (N57Q, K51Q) containing CAG-μDys intramuscularly in vivo, which demonstrated a significant improvement in grip strength 18 weeks after gene therapy. Subsequent evaluation of a codon-optimised microdystrophin transgene under the control of the optimal CAG promoter and capsid (AAV9K51Q) by intramuscular administration revealed enhanced muscle grip strength and dystrophin-glycoprotein complex restoration up to 4 months after gene therapy. Based on the improved performance of AAV9K51Q vectors during intramuscular gene transfer, we performed a systemic administration of these vectors alone, and a comparison with the control group revealed a significantly increased muscle contraction force by 1.6-1.7 fold and a 30%-60% dystrophin restoration in skeletal and cardiac muscles, up to 14 months after gene therapy. Collectively, our study underscores the therapeutic potential of engineered AAV9 vectors for potential clinical application in patients with DMD.

Indexed as

DependovirusDystrophinGenetic TherapyGenetic VectorsMuscular Dystrophy, DuchenneAnimalsDisease Models, AnimalGene Therapy AgentsHumansMiceMice, Inbred mdxMuscle, SkeletalPhenotypePromoter Regions, GeneticTransgenesDystrophinAAV9codon‐optimised microdystrophinDuchenne muscular dystrophygene therapy

Identifiers

PMID41793215
PMCPMC12966934

What OpenQuestion holds

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LicenceCC BY
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Registered trials

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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.