CT-101 enhancesγ-globin gene transcription in SCD mice.

<p>To determine the ability of CT-101 to induce HbF in SCD mice, flow cytometry and RT-qPCR analyses were completed in week 0 for n = 12 mice per treatment group. Deaths were observed during the study, thus by week 4 we evaluated water (n = 12), hydroxyurea (HU; n = 10), FORM-3 (Veh; n = 10),...

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التفاصيل البيبلوغرافية
المؤلف الرئيسي:	Mayuko Takezaki (12700243) (author)
مؤلفون آخرون:	Biaoru Li (628604) (author), Hongyan Xu (29047) (author), Nikhil Patel (2288824) (author), Rudolf Lucas (402664) (author), Ryan E. Cerbone (21354826) (author), Sivanagireddy Koti (21354829) (author), Clifford L. Hendrick (21354832) (author), Louis H. Junker (21354835) (author), Betty S. Pace (12700246) (author)
منشور في:	2025
الموضوعات:	Medicine Cell Biology Genetics Molecular Biology Neuroscience Pharmacology Developmental Biology Cancer Hematology similar drug uptake sickle cell disease production occurs leading townes scd mice scd mouse treated hbf protein synthesis fetal hemoglobin expression chemotherapeutic hbf inducers approved fetal hemoglobin subsequent confirmatory studies subsequent studies xlink "> unmet need united states study aimed shortened lifespan point mutation pharmacokinetic parameters peripheral blood organ damage optimal dose occlusive pain novel agent hemorrhagic infarcts findings support demonstrating increases clinical testing clinical severity
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CT-101 enhancesγ-globin gene transcription in SCD mice.

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