Curing Hemophilia: Repeated Treatments versus a One-Off Fix

Mol Ther. 2020 May 6;28(5):1229-1230. doi: 10.1016/j.ymthe.2020.04.012. Epub 2020 Apr 17.

Authors

Ning Li¹, Radoslaw Kaczmarek²

Affiliations

¹ Herman B Wells Center for Pediatric Research, Indiana University, Indianapolis, IN 46202, USA.
² Herman B Wells Center for Pediatric Research, Indiana University, Indianapolis, IN 46202, USA; Hirszfeld Institute of Immunology and Experimental Therapy, Polish Academy of Sciences, Wroclaw, Poland. Electronic address: rkaczmar@iu.edu.

No abstract available

Publication types

Editorial

MeSH terms

Antibodies, Bispecific / therapeutic use*
Antibodies, Monoclonal, Humanized / therapeutic use*
Cell- and Tissue-Based Therapy / methods*
Drug Delivery Systems / methods*
Factor VIII / genetics
Factor VIII / pharmacokinetics
Factor VIII / therapeutic use*
Gene Editing
Genetic Therapy / methods*
Half-Life
Hemophilia A / drug therapy*
Humans
Immunotherapy / methods*
Treatment Outcome

Substances

Antibodies, Bispecific
Antibodies, Monoclonal, Humanized
emicizumab
Factor VIII