A step toward stem cell engineering in Vivo

“Breda et al. [Children’s Hospital of Philadelphia] designed a strategy to reprogram bone marrow stem cells directly within the body without the need for donor cells or the use of potentially toxic conditioning regimens such as chemotherapy or radiation . . . . Messenger RNA was delivered to bone marrow stem cells by intravenous injection in lipid nanoparticles, facilitating both gene editing and bone marrow transplantation. The ability to engineer bone marrow cells inside a patient without the need for traditional transplantation approaches could hold promise for a number of genetic disorders.”     MORE

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