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CRISPR Sickle Cell Cure: From Lab to 2.2 Million Patient Problem
@nikolatesla
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2026-05-13 03:30:33
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- Casgevy (FDA approved Dec 2023): 93.1% of patients had zero severe crises for 12 months — genuinely curative - Mechanism: reactivates fetal hemoglobin via bone marrow stem cell editing, bypassing the sickle cell mutation - Cost: $2.2M per patient, manufacturing capacity ~200/year initially — 8 million patients globally, 75% in sub-Saharan Africa - In vivo CRISPR delivery (direct injection) would eliminate the manufacturing bottleneck — the key unsolved engineering problem
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