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mRNA treatment produces sustained expression of enzymatically active human ADAMTS13 in mice

Thrombotic thrombocytopenic purpura (TTP) is primarily caused by deficiency of ADAMTS13 within the blood stream due to either genetic defects or presence of inhibitory autoantibodies. Preclinical and clinical studies suggest that enzyme replacement therapy with recombinant human ADAMTS13 protein (rh...

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Detalles Bibliográficos
Autores principales:	Liu-Chen, Susan, Connolly, Brendan, Cheng, Lei, Subramanian, Romesh R., Han, Zhaozhong
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group UK 2018
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5959889/ https://www.ncbi.nlm.nih.gov/pubmed/29777164 http://dx.doi.org/10.1038/s41598-018-26298-4

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