ReviewMolecular therapy. Methods & clinical development2025
Stem cell and gene therapies for leukodystrophies.
Review in Molecular therapy. Methods & clinical development, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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
1 citing paper in PubMed.
- Glial Ion Channels in Myelin Pathophysiology: Insights from Leukodystrophies.Life (Basel, Switzerland) · 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
Leukodystrophies form a group of rare genetic disorders characterized by the progressive degeneration of white matter in the brain, often presenting in childhood with life-threatening consequences. Current therapeutic options are limited, particularly after symptom onset, and delayed diagnosis exacerbates disease progression. Stem cell and gene therapies have emerged as promising strategies to address these challenges. Neural stem cells (NSCs) and hematopoietic stem cells (HSCs) offer potential for CNS repair through differentiation into neurons and/or glial cells. Human pluripotent stem cells (hPSCs) provide a valuable model for studying disease mechanisms, drug screening, and therapeutic development. Additionally, mesenchymal stem cells (MSCs) exhibit immunomodulatory and secretory properties that may confer therapeutic benefits. Advanced gene therapies, including stem cell-based gene therapy and adeno-associated virus (AAV)-mediated gene replacement therapy (GRT), hold promise for curative interventions. This review highlights recent advances and challenges in stem cell and gene therapies for leukodystrophies, proposing a strategic integration of these approaches to maximize therapeutic efficacy by addressing their respective strengths and limitations.
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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 OpenQuestion graph.