TY - JOUR

T1 - Endogenous factor VIII synthesis from the intron 22-inverted F8 locus may modulate the immunogenicity of replacement therapy for hemophilia A

AU - Pandey, Gouri Shankar

AU - Yanover, Chen

AU - Miller-Jenkins, Lisa M.

AU - Garfield, Susan

AU - Cole, Shelley A.

AU - Curran, Joanne E.

AU - Moses, Eric K.

AU - Rydz, Natalia

AU - Simhadri, Vijaya

AU - Kimchi-Sarfaty, Chava

AU - Lillicrap, David

AU - Viel, Kevin R.

AU - Przytycka, Teresa M.

AU - Pierce, Glenn F.

AU - Howard, Tom E.

AU - Sauna, Zuben E.

AU - Lusher, Jeanne

AU - Chitlur, Meera

AU - Ameri, Afshin

AU - Natrajan, Kavita

AU - Iyer, Rathi V.

AU - Thompson, Alexis A.

AU - Watts, Raymond G.

AU - Kempton, Christine L.

AU - Kessler, Craig

AU - Barrett, John C.

AU - Martin, Erica J.

AU - Key, Nigel

AU - Kruse-Jarres, Rebecca

AU - Lessinger, Cindy

AU - Pratt, Kathleen P.

AU - Josephson, Neil

AU - McRedmond, Kevin

AU - Withycombe, Janice

AU - Walsh, Christopher

AU - Matthews, Dana

AU - Mahlangu, Johnny

AU - Krause, Amanda

AU - Schwyzer, Rosemary

AU - Thejpal, Rajendra

AU - Rapiti, Nadine

AU - Goga, Yasmin

AU - Coetzee, Marius

AU - Stones, David

AU - Mann, Kenneth

AU - Butenas, Saulius

AU - Almasy, Laura

AU - Blangero, John

AU - Carless, Mel

AU - Raja, Rajalingam

AU - Reed, Elaine

PY - 2013/10

Y1 - 2013/10

N2 - 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