New sickle cell research involves gene-editing technology to target and edit human blood stem cells
Danielle Lee of Garfield Heights demonstrates karate moves during a class at Golden Pyramid Martial Arts Center in Maple Heights. Lee, a sickle cell patient, joined the class after a Cleveland Clinic trial investigating gene editing as a treatment for the disease lessened her symptoms.CLEVELAND, Ohio — Like many young women, Danielle Lee often makes decisions that worry her elders.
“I know sickle cell — I know what that brings,” Lee recalled thinking. “But with this trial, it’s an experiment. What if I end up worse off than before and can’t pursue my dreams? Because I don’t want to sit in the house and be depressed and feel like I can’t do anything.”Abnormal genetic code behind sickle cell
“Think of this as like a full textbook, and one letter change makes our red blood cells change shape from being circular and flexible, to rigid and sickle shaped,” said CRISPR and Caspase 12 together are able to make precise genome editing leading to permanent changes in the DNA of living organisms.These gene-editing technologies target the part of the gene that produces fetal hemoglobin, enabling the body to produce high levels of fetal hemoglobin, Hanna said.
Other similar clinical trials that are closer to completion may deliver gene therapy treatments to sickle cell patients as soon as next year, Hanna said.
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