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Sustained Genetic Silencing of BCL11A Using shmiR Vector in Sickle Cell Disease: Long-Term Safety and Efficacy
Hematology-Oncologygene therapyBCL11A

Sustained Genetic Silencing of BCL11A Using shmiR Vector in Sickle Cell Disease: Long-Term Safety and Efficacy

By MedXY|Aug 24, 2026

This article reviews the first-in-human study demonstrating durable and safe posttranscriptional silencing of BCL11A in sickle cell disease patients using a shmiR vector, resulting in stable fetal hemoglobin induction and clinical benefit o

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