A multitude of tools now exist that allow us to precisely manipulate the human genome in a myriad of different ways. However, successful delivery of these tools to the cells of human patients remains a major barrier to their clinical implementation. Here we introduce a new cellular approach for genetic engineering, ecreted article nformation ransfer (SPIT) that utilizes human cells as delivery vectors for genetic engineering.
View Article and Find Full Text PDFSome gene polymorphisms can lead to monogenic diseases, whereas other polymorphisms may confer beneficial traits. A well-characterized example is congenital erythrocytosis-the non-pathogenic hyper-production of red blood cells-that is caused by a truncated erythropoietin receptor. Here we show that Cas9-mediated genome editing in CD34 human haematopoietic stem and progenitor cells (HSPCs) can recreate the truncated form of the erythropoietin receptor, leading to substantial increases in erythropoietic output.
View Article and Find Full Text PDFA multitude of tools now exist that allow us to precisely manipulate the human genome in a myriad of different ways. However, successful delivery of these tools to the cells of human patients remains a major barrier to their clinical implementation. Here we introduce a new cellular approach for genetic engineering, ecreted article nformation ransfer (SPIT) that utilizes human cells as delivery vectors for genetic engineering.
View Article and Find Full Text PDFLancet Microbe
January 2024