ArticleMolecular therapy : the journal of the American Society of Gene Therapy2024
In vivo base editing of a pathogenic Eif2b5 variant improves vanishing white matter phenotypes in mice.
Article 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 7 papers.
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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.
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Who cites it
7 citing papers in PubMed, 5 citations in OpenAlex.
- Base editing for precision therapeutics.Cell genomics · 2026Review
- Prime editing of a pathogenicScience translational medicine · 2026Article
- Article
- Long-read sequencing and next-generation CRISPR editors: a unified pipeline for rare disease precision medicine with ethical and regulatory perspectives.Frontiers in medicine · 2026Review
- Prime editing of the βNature communications · 2025Article
- Review
- Review
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Authors and funding
11 authors at 3 institutions in 3 countries.
Funding
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Abstract
Vanishing white matter (VWM) is a fatal leukodystrophy caused by recessive mutations in subunits of the eukaryotic translation initiation factor 2B. Currently, there are no effective therapies for VWM. Here, we assessed the potential of adenine base editing to correct human pathogenic VWM variants in mouse models. Using adeno-associated viral vectors, we delivered intein-split adenine base editors into the cerebral ventricles of newborn VWM mice, resulting in 45.9% ± 5.9% correction of the Eif2b5
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Registered trials
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