Gene Silencing of BCL11A Increases Fetal Hemoglobin in Patients with Sickle Cell Disease

This small pilot study validates use of this gene therapy.

While allogeneic transplantation can cure sickle cell disease, complication rates are higher in adults than in children, and donor availability is a limiting factor. Two recently approved drugs, voxelate and crizanlizumab, can modify the clinical course, but neither definitively treats the disease. Therefore, novel strategies are required, and a key target is induction of fetal hemoglobin (HbF), as increases in HbF are associated with a favorable clinical course.

To induce fetal hemoglobin production, investigators utilized a lentiviral vector (BCH-BB694) to knock down BCL11A, a transcription factor that suppresses fetal hemoglobin, via RNA interference. In a single-center, open-label pilot study, six patients (ages 7–25 years) underwent col…

Reviewing Author

Brady L. Stein, MD, MHS

Disclosures

Leadership Positions in Professional SocietiesAmerican Society of Hematology (Senior Editor, ASH Self-Assessment Program; guideline development)

Brady L. Stein, MD, MHS

Disclosures

Leadership Positions in Professional SocietiesAmerican Society of Hematology (Senior Editor, ASH Self-Assessment Program; guideline development)

Gene Silencing of BCL11A Increases Fetal Hemoglobin in Patients with Sickle Cell Disease

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Reviewing Author