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Trans-splicing vectors expand the utility of adeno-associated virus for gene therapy

Adeno-associated viral (AAV) vectors have demonstrated considerable promise for gene therapy of inherited diseases. However, with a packaging size of <5 kb, applications have been limited to relatively small disease genes. Based on the finding that AAV genomes undergo intermolecular circular conc...

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Autores principales: Yan, Ziying, Zhang, Yulong, Duan, Dongsheng, Engelhardt, John F.
Formato: Artigo
Lenguaje:Inglês
Publicado: National Academy of Sciences 2000
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Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC18714/
https://ncbi.nlm.nih.gov/pubmed/10841568
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