Pilot Feasibility Study of BCL11A as Gene Therapy Target in Sickle Cell Disease

December 7-10, 2019; Orlando, Florida
Preliminary results from pilot feasibility study suggest that a gene therapy construct targeting BCL11A is safe, effectively induces HbF expression, and improves sickling signs and symptoms when delivered to patients with severe SCD.
Format: Microsoft PowerPoint (.ppt)
File Size: 192 KB
Released: December 13, 2019

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