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Gene therapy and gene editing in sickle cell disease SeptemberModified December Several strategies are under study for individuals with sickle cell disease SCD who are seeking curative therapy. In a previous study, three individuals with SCD who were treated with a different gene editing approach disrupting the gamma globin promotor had marked reduction in vaso-occlusive complications and continue reading requirements [ 34,35 ].

All current gene therapy and gene editing approaches for SCD require autologous hematopoietic cell transplantation with myeloablative conditioning.

See "Investigational therapies for sickle cell disease", section on 'Gene therapy and gene editing'. Response rates were similar irrespective of prior exposure to venetoclax or presence how to get health insurance in ky BTK C mutations, which frequently inn upon progression on a covalent BTK inhibitor. We consider pirtobrutinib an option in this setting.

See "Treatment of relapsed or refractory chronic lymphocytic leukemia", section on 'Pirtobrutinib'.