1. Endogenous factor VIII synthesis from the intron 22-inverted F8 locus may modulate the immunogenicity of replacement therapy for hemophilia A
- Author
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Pandey, GS, Yanover, C, Miller-Jenkins, LM, Garfield, S, Cole, SA, Curran, JE, Moses, EK, Rydz, N, Simhadri, V, Kimchi-Sarfaty, C, Lillicrap, D, Viel, KR, Przytycka, TM, Pierce, GF, Howard, TE, Sauna, ZE, Lusher, J, Chitlur, M, Ameri, A, Natarajan, K, Iyer, RV, Thompson, AA, Watts, RG, Kempton, CL, Kessler, C, Barrett, JC, Martin, EJ, Key, N, Kruse-Jarres, R, Lessinger, C, Pratt, KP, Josephson, N, McRedmond, K, Withycombe, J, Walsh, C, Matthews, D, Mahlangu, J, Krause, A, Schwyzer, R, Thejpal, R, Rapiti, N, Goga, Y, Coetzee, M, Stones, D, Mann, K, Butenas, S, Almasy, L, Blangero, J, Carless, M, Raja, R, and Reed, E
- Subjects
congenital, hereditary, and neonatal diseases and abnormalities ,hemic and lymphatic diseases - Abstract
Neutralizing antibodies (inhibitors) to replacement factor VIII (FVIII, either plasma derived or recombinant) impair the effective management of hemophilia A. Individuals with hemophilia A due to major deletions of the FVIII gene (F8) lack antigenically cross-reactive material in their plasma ("CRM-negative"), and the prevalence of inhibitors in these individuals may be as high as 90%. Conversely, individuals with hemophilia A caused by F8 missense mutations are CRM-positive, and their overall prevalence of inhibitors is
- Published
- 2013