High-potency MyoAAV capsids enhanced skeletal muscle correction in a mouse model of GSD IIIa
Glycogen storage disease type IIIa (GSD IIIa) affects multiple tissues, including liver, heart, and skeletal muscles. We recently reported that an adeno-associated virus serotype 9 vector expressing pullulanase, a bacterial glycogen debranching enzyme, driven by an immunotolerizing dual promoter (AA...
Kaydedildi:
| Asıl Yazarlar: | , , , , , , , |
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| Materyal Türü: | Artigo |
| Dil: | Inglês |
| Baskı/Yayın Bilgisi: |
Elsevier
2025-09-01
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| Seri Bilgileri: | Molecular Therapy: Methods & Clinical Development |
| Konular: | |
| Online Erişim: | http://www.sciencedirect.com/science/article/pii/S2329050125001627 |
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