Genetic Engineering Publications - GEG Tech top picks

Gene therapy that alters hemoglobin genes may be an answer to curing sickle cell disease (SCD) and beta thalassemia. These two common life-threatening anemias afflict millions of individuals across the globe. Scientists at St. Jude Children's Research Hospital and the Broad Institute of MIT and Harvard used a next-generation genome editing technology, adenosine base editing, to restart fetal hemoglobin expression in SCD patient cells. The approach raised the expression of fetal hemoglobin to higher, more stable, and more uniform levels than other genome editing technologies that use CRISPR/Cas9 nuclease in human hematopoietic stem cells. The findings were published in Nature Genetics. 

Sickle cell disease is a genetic blood disorder that affects the structure and function of hemoglobin. This disease affects millions of people worldwide. It reduces the ability of red blood cells to carry oxygen efficiently and progresses to chronic vascular disease. The disease is caused by a single letter mutation in human DNA at a gene in beta-globulin. To combat this disease, researchers at UC San Francisco, UC Berkeley and UCLA have developed a CRISPR-based gene correction therapy, named CRISPR_SCD001, for patients with sickle cell disease. This therapy is based on the use of blood stem cells taken from patients that are modified with a CRISPR-Cas9 nuclease introduced into the cells by electroporation in order to stimulate repair of the sickle cell mutation that causes the patients to form deformed red blood cells. The modified stem cells are then reintroduced into the patient. This is the first-time researchers will attempt to correct the beta-globulin gene in patients' own cells using non-viral delivered CRISPR tools. Their therapy has received approval from the U.S. Food and Drug Administration for an initial human clinical trial.

The study will last 4 years and will include 6 adults and 3 adolescents with severe sickle cell disease.