# Editorial for Special Issue on Gene Therapy of Rare Diseases

**Authors:** Cord Brakebusch

PMC · DOI: 10.3390/cells15060504 · 2026-03-12

## Full-text entities

- **Genes:** Galc (galactosylceramidase) [NCBI Gene 14420] {aka 2310068B06Rik, A930008M05Rik, Gacy, twi, twitcher}
- **Diseases:** beta-thalassemia (MESH:D017086), rare diseases (MESH:D035583), injury to (MESH:D014947), alpha1-antitrypsin disease (MESH:D019896), spinal muscular atrophy (MESH:D009134), metachromatic leukodystrophy (MESH:D007966), cerebral adrenoleukodystrophy (MESH:D000326), Diamond-Blackfan Anemia (MESH:D029503), lysosomal storage disorder (MESH:D016464), Globoid Cell Leukodystrophy (MESH:D007965), hemophilia A and B. (MESH:D002836), sickle cell anemia (MESH:D000755), Wilson disease (MESH:D006527), of the liver (MESH:D017093)
- **Chemicals:** AAV9 (-)
- **Species:** Mus musculus (house mouse, species) [taxon 10090], Homo sapiens (human, species) [taxon 9606]

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Source: https://tomesphere.com/paper/PMC13025261