Scientists reprogram blood cells with fat balls that target bone marrow

Some people with sickle cell disease are now healthy thanks to an experimental treatment: Researchers edit the DNA of their defective blood stem cells in a lab dish, then infuse the cells back into the body. This “ex vivo” procedure is costly and complex, however, putting it out of reach for many patients in poor countries. Now, scientists have shown they can reprogram blood cells directly inside the body by targeting genetically loaded balls of fat to the bone marrow. The advance, described today in Science , only works in mice so far and hasn’t yet cured a disease in the animals. But if it translates to humans, it could provide a quicker, cheaper way to treat sickle cell and other blood diseases in people, says study author Hamideh Parhiz, a pharmaceutical biotechnologist at the University of Pennsylvania (UPenn). Scientists have successfully tackled many genetic disorders with gene therapy—packaging normal versions of genes into viruses to replace the defective ones in the body . They’re also ferrying into cells components of gene editors to repair genes. But to do this for blood disorders, the cells must be removed from the body and genetically modified in a lab dish. Another problem is that ex vivo approaches typically require chemotherapy to wipe out the patient’s defective blood cells—a grueling and risky procedure. Recently, researchers have treated sickle cell disease in mice by using viruses to ...
Source: Science of Aging Knowledge Environment - Category: Geriatrics Source Type: research