A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells

Nine dogs with hemophilia A were treated with adeno-associated viral (AAV) gene therapy and followed for up to 10 years. Administration of AAV8 or AAV9 vectors expressing canine factor VIII (AAV-cFVIII) corrected the FVIII deficiency to 1.9–11.3% of normal FVIII levels. In two of nine dogs, levels o...

Ausführliche Beschreibung

Gespeichert in:

Bibliographische Detailangaben
Veröffentlicht in:	Nature biotechnology 2021-01, Vol.39 (1), p.47-55
Hauptverfasser:	Nguyen, Giang N., Everett, John K., Kafle, Samita, Roche, Aoife M., Raymond, Hayley E., Leiby, Jacob, Wood, Christian, Assenmacher, Charles-Antoine, Merricks, Elizabeth P., Long, C. Tyler, Kazazian, Haig H., Nichols, Timothy C., Bushman, Frederic D., Sabatino, Denise E.
Format:	Artikel
Sprache:	eng
Schlagworte:	631/114/2398 631/114/794 631/1647/767/1424 631/208/199 631/61/201 Agriculture Animals Bioinformatics Biomedical and Life Sciences Biomedical Engineering/Biotechnology Biomedicine Biotechnology Care and treatment Coagulation factors Deoxyribonucleic acid Dependovirus - genetics DNA Dogs Expression vectors Factor VIII - genetics Factor VIII - metabolism Factor VIII deficiency Gene therapy Genetic Therapy - veterinary Genotoxicity Hemophilia Hemophilia A - therapy Hemophilia A - veterinary Hepatocytes Hepatocytes - metabolism Integration Life Sciences Liver Liver - cytology Liver - metabolism Liver - physiopathology Medical research Medicine, Experimental Methods Prospective Studies Tumors Vectors (Biology)
Online-Zugang:	Volltext
Tags:	Tag hinzufügen Keine Tags, Fügen Sie den ersten Tag hinzu!

Schreiben Sie den ersten Kommentar!