A Method To Cure Sickle Cell Disease

Patent Status

Brief Description

Sickle cell disease is an inherited recessive disease, caused by a single nucleotide polymorphism in Beta-globin (HBB). The modified hemoglobin causes normally round red blood cells to take on a sticky, sickle-shaped form. Sickle red blood cells clog blood vessels, causing acute pain “crises” and vasculopathy. Additional complications and consequences associated with sickle cell disease include organ damage, organ failure, increased risk of stroke, pulmonary hypertension, acute chest syndrome (ACS), and decreased lifespan. There is no widely available cure for sickle cell disease. Treatments include allogeneic bone marrow transplants, which can be risky and limited by donor availability.

UC Berkeley researchers and others have created a method of modifying a globin gene in the genome of a hematopoietic stem/progenitor cell (HSPCs) by obtaining HSPCs from an individual with a globin gene having a sickle cell disease (SCD)-associated single nucleotide polymorphism (SNP) to generate an in vitro population of CD34+ HSPCs and then contacting the in vitro population with a genome editing composition, as described in further detail below. 

Suggested uses

• therepeutic treatment of sickle cell disease