From lipid nanoparticles to precision therapies for hematologic diseases: getting closer

Modification of hematopoietic stem cells (HSCs), in both molecular and cellular terms, has been a long-pursued goal in experimental and clinical hematology due to the key roles that such cells play in the production of blood cells (hematopoiesis) and in the pathogenesis of several hematologic diseases. In a recent article published in Science, Breda et al1describe a novel method for the genetic modification of HSCs. This new experimental approach, based on the use of lipid nanoparticles (LNPs), represents an important step forward in the development of genetic and cellular therapies for hematologic diseases.