One Shot Gene Therapy for Sickle Cell Disease
October 10, 2023 | Terry Sharrer
“On July 27 in Science, scientists from Children’s Hospital of Philadelphia and the University of Pennsylvania’s Perelman School of Medicine revealed a new proof-of-concept mRNA-based in vivo gene therapy that is meant to correct the gene mutation that causes sickle cell in human cells without needing to alter cells outside the body. “The bottom line is that right now, if you want to do gene therapy—especially for stem cells in the bone marrow—you need to basically perform three steps: take the cells out, modify them, and put them back,”. . . . The advantage of our approach is that you could eventually modify the cells genetically with a single injection.” MORE
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