Researchers at the Children's Hospital of Philadelphia and the Perelman School of Medicine at the University of Pennsylvania have developed a proof-of-concept model for delivering gene-editing tools to treat blood disorders. Their approach uses mRNA encapsulated in lipid nanoparticles as a technology platform to carry out in vivo cellular reprogramming, modifying diseased blood cells directly within the body. Use of this platform in clinical settings could expand access and reduce the cost of gene therapies for blood disorders, many of which currently require chemotherapy and a stem cell transplant.
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