Gene therapy for the leukodystrophies: From preclinical animal studies to clinical trials
Leukodystrophies are progressive single gene disorders affecting the white matter of the brain. Several gene therapy trials are in progress to address the urgent unmet need for this patient population. We performed a comprehensive literature review of all gene therapy clinical trials listed in www.c...
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| Hauptverfasser: | , , , |
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| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
Elsevier
2024-07-01
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| Schriftenreihe: | Neurotherapeutics |
| Schlagworte: | |
| Online-Zugang: | http://www.sciencedirect.com/science/article/pii/S1878747924001302 |
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