abstract |
A method for producing a hematopoietic progenitor cell that has decreased mRNA or BCL11A protein expression, the method comprises contacting an isolated hematopoietic progenitor cell ex vivo or in vitro with at least one DNA-directed endonuclease or a vector carrying the coding sequence of a DNA-directed endonuclease so that the DNA-directed endonuclease cleaves the genomic DNA of the cell at the 60,716,189-60,728,612 location of chromosome 2 (according to the hg 19 human genome assembly of the UCSC genomic navigator) , to thereby reduce mRNA or protein expression of BCL11A. |