CRISPR/Cas9 Gene Editing for Curing Sickle Cell Disease

Sickle cell disease (SCD) is the most common monogenic blood disorder marked by severe pain, end-organ damage, and early mortality. Treatment options for SCD remain very limited. There are only three FDA approved drugs to reduce acute complications. The only curative therapy for SCD is hematopoietic stem cell transplantation, typically from a matched, related donor. Ex vivo engineering of autologous hematopoietic stem and progenitor cells followed by transplantation of genetically modified cells potentially provides a permanent cure applicable to all patients regardless of the availability of suitable donors and graft-vs-host disease.

Source: Transfusion and Apheresis Science - January 10, 2021 Category: Hematology Authors: So Hyun Park, Gang Bao Source Type: research