Engineering and In Vitro Selection of a Novel AAV3B Variant with High Hepatocyte Tropism and Reduced Seroreactivity

Limitations to successful gene therapy with adeno-associated virus (AAV) can comprise pre-existing neutralizing antibodies to the vector capsid that can block cellular entry, or inefficient transduction of target cells that can lead to sub-optimal expression of the therapeutic transgene. Recombinant...

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Publicado en:Mol Ther Methods Clin Dev
Autores principales: Biswas, Moanaro, Marsic, Damien, Li, Ning, Zou, Chenhui, Gonzalez-Aseguinolaza, Gloria, Zolotukhin, Irene, Kumar, Sandeep R.P., Rana, Jyoti, Butterfield, John S.S., Kondratov, Oleksandr, de Jong, Ype P., Herzog, Roland W., Zolotukhin, Sergei
Formato: Artigo
Lenguaje:Inglês
Publicado: American Society of Gene & Cell Therapy 2020
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Original Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC7591349/
https://ncbi.nlm.nih.gov/pubmed/33145371
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.09.019
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