abstract |
An ex vivo or in vitro method for increasing fetal hemoglobin levels in a cell, the method comprising the steps of contacting a hematopoietic progenitor cell with an effective amount of a composition comprising a BCL11A inhibitor, thereby increasing the expression of fetal hemoglobin in said cell, or its progeny, in relation to said cell before said contacting, wherein the BCL11A inhibitor is a BCL11A specific nucleic acid that inhibits the expression of BCL11A, and where the nucleic acid BCL11A specific is an interference RNA agent specific to BCL11A, a vector encoding an interference RNA agent specific to BCL11A, an antisense construct or molecule, or a ribozyme. |