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Rational engineering of a functional CpG-free ITR for AAV gene therapy

Inverted terminal repeats (ITRs) are the only wild-type components retained in the genome of adeno-associated virus (AAV) vectors. To determine whether ITR modification is a viable approach for AAV vector engineering, we rationally deleted all CpG motifs in the ITR and examined whether CpG eliminati...

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Detalles Bibliográficos
Autores principales: Pan, Xiufang, Yue, Yongping, Boftsi, Maria, Wasala, Lakmini P., Tran, Ngoc Tam, Zhang, Keqing, Pintel, David J., Tai, Phillip W. L., Duan, Dongsheng
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8983793/
https://www.ncbi.nlm.nih.gov/pubmed/34611321
http://dx.doi.org/10.1038/s41434-021-00296-0