Life-altering treatments could soon be available for sickle cell, but questions remain.
has been defined by pain. He was born with sickle cell disease, which causes red blood cells to be sticky and C-shaped, not smooth and round. These cells are supposed to move freely through blood vessels, carrying oxygen to the body. But in people with this inherited form of anemia, they clump together and block blood flow. This triggers excruciating episodes known as pain crises, which can happen anywhere in the body and last for hours or even weeks.
As an adult, it didn’t get easier. Sometimes he could ward off the pain with ibuprofen and get back to work the next day. But every few months, a severe crisis sent him to the hospital. Things got so bad that in 2019, he enrolled in a clinical trial at the University of California, Los Angeles, which has been testing a gene therapy to cure sickle cell. It involves genetically modifying patients’ blood-forming stem cells in the lab so that they can produce healthy red blood cells.
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