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

• المؤلفون الرئيسيون: Kazuhiro Muramatsu (مؤلف), Shin-ichi Muramatsu (مؤلف)
• التنسيق: كتاب
• منشور في: Elsevier, 2023-02-01T00:00:00Z.
• الموضوعات: article
• الوصول للمادة أونلاين: Connect to this object online.

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...