ReviewMolecular therapy : the journal of the American Society of Gene Therapy2024
Biomarkers for gene therapy clinical trials of lysosomal storage disorders.
Review in Molecular therapy : the journal of the American Society of Gene Therapy, 2024. 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
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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.
- A multi-omics-empowered framework for precision diagnosis and treatment of lysosomal diseases.Journal of pharmaceutical analysis · 2025Review
- The Emerging Roles of Vacuolar-Type ATPase-Dependent Lysosomal Acidification in Cardiovascular Disease.Biomolecules · 2025Review
- Staying on target in gene and cell therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Lysosomal storage disorders (LSDs) are multisystemic progressive disorders caused by defects in proteins involved in lysosomal function. Different gene therapy strategies are under clinical investigation in several LSDs to overcome the limitations of available treatments. However, LSDs are slowly progressive diseases that require long-term studies to establish the efficacy of experimental treatments. Biomarkers can be reliable substitutes for clinical responses and improve the efficiency of clinical trials, especially when long-term disease interventions are evaluated. In this review, we summarize both available and future biomarkers for LSDs and discuss their strengths and weaknesses.
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Registered trials
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