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Article

Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease

5 Dec 2020

Abstract excerpt

BACKGROUND: is a repressor of γ-globin expression and HbF production in adult erythrocytes. Its down-regulation is a promising therapeutic strategy for induction of HbF. METHODS: mRNA embedded in a microRNA (shmiR), allowing erythroid lineage-specific knockdown. Patients were assessed for primary end points of engraftment and safety and for hematologic and clinical responses to treatment. RESULTS: As of October...

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PMID: 33283990
DOI: 10.1056/nejmoa2029392

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