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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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| Publicado no: | Sci Rep |
|---|---|
| Main Authors: | , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Nature Publishing Group UK
2018
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5959889/ https://ncbi.nlm.nih.gov/pubmed/29777164 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41598-018-26298-4 |
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