December 5, 2020Open Access

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

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Abstract

This study validates BCL11A inhibition as an effective target for HbF induction and provides preliminary evidence that shmiR-based gene knockdown offers a favorable risk-benefit profile in sickle cell disease. (Funded by the National Institutes of Health; ClinicalTrials.gov number, NCT03282656).

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

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Abstract

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