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Adeno-associated virus vector-based gene therapies for pediatric diseases

30 Sept 2022

Abstract excerpt

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 therapy using the AAV9 vector, which crosses the blood-brain barrier, has been performed in more than 2000 patients...

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PMID: 36266189
DOI: 10.1016/j.pedneo.2022.09.004

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