Determining the Minimally Effective Dose of a Clinical Candidate Adeno-Associated Virus Vector in a Mouse Model of Hemophilia A

Hemophilia A, a bleeding disorder, affects 1:5,000 males and is caused by a deficiency of human blood coagulation factor VIII (hFVIII). Studies in mice and macaques identified AAVhu37.E03.TTR.hFVIIIco-SQ.PA75 as a clinical candidate gene therapy vector to treat hemophilia A. In this study, we sought...

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Detalles Bibliográficos
Autores principales: Greig, Jenny A., Smith, Melanie K., Nordin, Jayme M.L., Goode, Tamara, Chroscinski, Edward A., Buza, Elizabeth L., Schmidt, Nicole, Kattenhorn, Lisa M., Wadsworth, Samuel, Wilson, James M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2022
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9063151/
https://www.ncbi.nlm.nih.gov/pubmed/34652966
http://dx.doi.org/10.1089/hum.2021.108

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