Adeno-associated virus vector-based gene therapies for pediatric diseases
Gene therapy using adeno-associated virus (AAV) is a rapidly developing technology with widespread treatment potential. AAV2 vectors injected directly into the brain by stereotaxic brain surgery have shown good results in treating aromatic l-amino acid decarboxylase deficiency. Moreover, gene therap...
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| Main Authors: | , |
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| Format: | Book |
| Published: |
Elsevier,
2023-02-01T00:00:00Z.
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