Using a Human Liver Tissue Equivalent (hLTE) Platform to Define the Functional Impact of Liver-Directed AAV Gene Therapy

Clinical trials employing AAV vectors for hemophilia A have been hindered by unanticipated immunological and/or inflammatory responses in some of the patients. Also, these trials have often yielded lower levels of transgene expression than were expected based upon preclinical studies, highlighting t...

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Veröffentlicht in:	Blood 2021-11, Vol.138 (Supplement 1), p.2938-2938
Hauptverfasser:	Ramamurthy, Ritu M, Zheng, Wen Ting, George, Sunil, Wan, Meimei, Zhou, Yu, Lu, Baisong, Bishop, Colin E, Atala, Anthony, Porada, Christopher D, Almeida-Porada, M. Graca
Format:	Artikel
Sprache:	eng
Online-Zugang:	Volltext
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