ReviewMolecular therapy : the journal of the American Society of Gene Therapy2026
Deaths in gene therapy of Duchenne muscular dystrophy and other diseases: Underlying mechanisms and mitigating strategies.
Review 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 3 papers.
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.
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.
Who cites it
3 citing papers in PubMed.
- Clinical immunogenicity in rAAV gene therapy: Insights and implications.Molecular therapy. Advances · 2026Review
- Article
- Spinal muscular atrophy: Biology, pathogenesis, and therapeutic advances.Therapeutic advances in neurological disorders · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors.
Funding
Abstract
Duchenne muscular dystrophy (DMD) is a fatal muscle degenerating disease caused by dystrophin deficiency. Adeno-associated virus (AAV)-based gene therapy holds promise for restoring missing dystrophin and improving quality of life. Many clinical trials have been conducted or are ongoing. Despite compelling preclinical data, the full potential of AAV gene therapy remains to be established in DMD patients. Importantly, high-dose intravenous AAV administration has resulted in hospitalizations and several deaths in patients afflicted by DMD and other inherited diseases due to innate and adaptive immune responses to the vectors. Although fatal outcomes are rare, a critical analysis of these cases may provide insights to refine systemic AAV gene therapy for DMD and other inherited diseases. Here, we review the clinical findings of the reported deaths and related cases in AAV gene therapy for DMD and other diseases. We also evaluate the underlying mechanisms and discuss mitigating strategies and future directions.
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What Socratic holds
Registered trials
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 Socratic graph.