ReviewRegenerative medicine2025
Developing a gene therapy for Charcot-Marie-Tooth disease: progress and challenges.
Review in Regenerative medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 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
5 citing papers in PubMed.
- AAV-mediated gene therapy demonstrates phenotypic rescue in a mouse model of Cockayne syndrome.The Journal of clinical investigation · 2026Article
- Spectrum of Hereditary Neuropathies in Adult Patients From Serbia.Journal of the peripheral nervous system : JPNS · 2026Article
- Peripheral Myelin Protein-22 and Its Prominence in Charcot-Marie-Tooth Disease.Chemical reviews · 2026Review
- PDXK-Related Neuropathy: A Case With a Novel Splice-Altering Missense Variant and Literature Review.Molecular genetics & genomic medicine · 2026Review
- Gene Therapy of Adrenomyeloneuropathy: Challenges, Target Cells, and Prospectives.Biomedicines · 2025Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Charcot-Marie-Tooth (CMT) disease refers to a diverse group of inherited and progressive neuropathies for which no approved treatments currently exist and management strategies remain limited to symptomatic interventions. Recent advances in gene therapy offer promising strategies to address CMT neuropathies. This review highlights key progress in developing gene silencing, replacement, or editing therapies for representative CMT types, and summarizes preclinical successes and translational challenges. Delivery technologies such as AAV vectors and nanoparticle systems have shown promise, but delivery limitations across the blood-nerve and -brain barriers, immune reactions and other potential toxicities, and scalability remain challenging. Advancing into the era of CMT treatments requires clinical readiness, which depends on optimizing therapeutic delivery, enhancing safety, and developing biomarkers for treatment monitoring.
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Identifiers
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.