Transplantation of CRISPR-CAS9 Corrected Hematopoietic Stem Cells (CRISPR_SCD001) in Patients with Severe Sickle Cell Disease

Statement of Benefit to California: 

The economic and health burdens of sickle cell disease are significant. As many as 13,000 persons in California are affected. The benefit of a curative therapy such as CRISPR_SCD001 is that it has the potential to be available in all affected persons if its safety and effectiveness is proved. This benefit will be magnified if it can be delivered safely early in life, before the chronic life-long problems of sickle cell disease have occurred.

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