Update on clinical gene therapy for hemophilia

In contrast to other diverse therapies for the X-linked bleeding disorder hemophilia that are currently in clinical development, gene therapy holds the promise of a lasting cure with a single drug administration. Near-to-complete correction of hemophilia A (factor VIII deficiency) and hemophilia B (...

Ausführliche Beschreibung

Bibliographische Detailangaben

Veröffentlicht in:	Blood 2019-01, Vol.133 (5), p.407-414
Hauptverfasser:	Perrin, George Q., Herzog, Roland W., Markusic, David M.
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Blood Coagulation Factors - genetics Dependovirus - genetics Genetic Therapy - methods Genetic Vectors - genetics Genetic Vectors - therapeutic use Hemophilia A - genetics Hemophilia A - therapy Hemophilia B - genetics Hemophilia B - therapy Humans New Therapeutics for Inherited and Acquired Bleeding Conditions Review Series
Online-Zugang:	Volltext
Tags:	Tag hinzufügen Keine Tags, Fügen Sie den ersten Tag hinzu!