Developing AAV-delivered nonsense suppressor tRNAs for neurological disorders
Adeno-associated virus (AAV)-based gene therapy is a clinical stage therapeutic modality for neurological disorders. A common genetic defect in myriad monogenic neurological disorders is nonsense mutations that account for about 11% of all human pathogenic mutations. Stop codon readthrough by suppre...
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| Main Authors: | Jiaming Wang, Guangping Gao, Dan Wang |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Elsevier
2024-07-01
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| Series: | Neurotherapeutics |
| Subjects: | |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S1878747924000771 |
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