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Endogenous factor VIII synthesis from the intron 22–inverted F8 locus may modulate the immunogenicity of replacement therapy for hemophilia A
- Pandey, Gouri Shankar;
- Yanover, Chen;
- Miller-Jenkins, Lisa M;
- Garfield, Susan;
- Cole, Shelley A;
- Curran, Joanne E;
- Moses, Eric K;
- Rydz, Natalia;
- Simhadri, Vijaya;
- Kimchi-Sarfaty, Chava;
- Lillicrap, David;
- Viel, Kevin R;
- Przytycka, Teresa M;
- Pierce, Glenn F;
- Howard, Tom E;
- Sauna, Zuben E
Published Web Location
https://doi.org/10.1038/nm.3270Abstract
Patients with hemophilia lacking functional coagulation factor VIII (FVIII) are treated with replacement FVIII proteins. The development of neutralizing antibodies to the replacement FVIII, a major clinical problem, depends on the nature of the mutation in the gene encoding FVIII. The authors find that a prevalent inversion allele can unexpectedly produce FVIII protein, explaining why individuals with this allele do not frequently develop neutralizing antibodies.
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