Current Therapies in Hemophilia: From Plasma-Derived Factor Modalities to CRISPR/Cas Alternatives.

Journal: The Tohoku Journal Of Experimental Medicine
Treatment Used: Current Therapies
Number of Patients: 0
MediFind Summary

Summary: This article discusses current therapies for the treatment of patients with hemophilia.

Conclusion: Current therapies for the treatment of patients with hemophilia include plasma-derived products and bioengineered recombinant factors VIII and IX, monoclonal antibodies, and gene therapy.


Since the middle of the last century, there have been amazing therapeutic advances for hemophilia such as the development of plasma-derived products and bioengineered recombinant factors VIII and IX (for hemophilia A and B, respectively) with improved stability, higher activity, and extended half-life. The recent use of a monoclonal antibody that mimics factor VIII activity (which is an efficient treatment for all hemophilia A phenotypes with or without inhibitors) has shown the great possibilities of non-factor therapies for improving the quality of life of hemophilia A patients, with a safer application and long-lasting effects. Gene therapy offers the promise of a "true cure" for hemophilia based on the permanent effect that a gene edition may render. Clinical trials developed in the last decade based on adenoviral vectors show modest but consistent results; now, CRISPR/Cas technology (which is considered the most efficient tool for gene edition) is being developed on different hemophilia models. Once the off-target risks are solved and an efficient switch on/off for Cas activity is developed, this strategy might become the most feasible option for gene therapy in hemophilia and other monogenic diseases.

Irving Jair Lara Navarro, Ana Rebeca Jaloma Cruz

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