Crouzier L, Richard EM, Sourbron J, Lagae L, Maurice T, Delprat B (2021) Use of zebrafish models to boost research in rare genetic diseases
For the second mouse study, antibody treatments were given subcutaneously on days 0, 4, 7, 14, and 21 of the study
Pharmacokinetic studies in rats All animal experiments were performed in compliance with standard operating procedures under a protocol approved by the Institutional Animal Care and Use Committee (IACUC) of Sungkyunkwan University (SKKUIACUC2025-06-29-1)
Most patients are asymptomatic with normal Hb and blood smear
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