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lentiviral vector targeting BCL11A
- Development stage
- Unknown
- Lead developer
- Boston Children's Hospital
- Modality
- Lentiviral Vectors → Retroviral Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
Overview
A lentiviral vector (LVV) designed for ex vivo gene therapy of sickle cell disease (SCD) that delivers an erythroid-specific microRNA-adapted short hairpin RNA (shRNAmiR) to knock down **BCL11A** expression in hematopoietic stem and progenitor cells (HSPCs). BCL11A is a transcriptional repressor of fetal hemoglobin (HbF); its knockdown reactivates HbF production while reducing sickle hemoglobin (HbS), inhibiting HbS polymerization and red blood cell sickling. Developed by researchers at Boston Children's Hospital (BCH) and UCLA, variants like **BCH-BB694** use a β-globin locus control region for lineage-specific expression, achieve high HbF levels (up to 70-88% pancellular), demonstrate low genotoxicity in preclinical assays, and support clinical-scale production with high titers for improved accessibility.
- Other names
- LCR-shRNAmiRbifunctional lentiviral vector targeting BCL11A and ZNF410
02
Targets
ZNF410 (Zinc finger protein 410)BCL11A (B-cell CLL/lymphoma 11A)
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