Method for predicting effectiveness of treatment of hemoglobinopathy

A method for treating hemoglobinopathy in an individual, which comprises a treatment step comprising:

• administering a second population of the modified CD34-positive HSPCs to the individual, wherein the modified CD34-positive HSPCs are derived from the individual and are modified to reduce BCL11A function (“modified TR cells”), and
• wherein the individual is selected for treatment based on the functional evaluation from an evaluation step which comprises:
• evaluating the ability of a first population of the modified CD34-positive hematopoietic stem cells/progenitor cells (“CD34-positive HSPCs”) to produce a desired level of γ-globin or fetal hemoglobin (HbF) after differentiation, wherein the modified CD34-positive HSPCs of the first population are derived from the individual and are modified to reduce BCL11A function (“modified EV cells”).

A method for determining whether an individual suffering from hemoglobinopathy is suitable or unsuitable for treating with a second population of the modified CD34-positive HSPCs (“modified TR cells”) derived from the individual and modified to reduce BCL11A function, wherein the method comprises an evaluation step comprising:

• evaluating the ability of a first population of the modified CD34-positive HSPCs to produce a desired level of γ-globin or fetal hemoglobin (HbF) after differentiation, wherein the modified CD34-positive HSPCs of the first population are derived from the individual and are modified to reduce BCL11A function (“modified EV cells”), and wherein if the first population of the modified CD34-positive HSPCs produce a desired level of γ-globin or fetal hemoglobin (HbF), the individual is suitable for treatment;
• wherein if the first population of the modified CD34-positive HSPCs do not produce a desired level of γ-globin or fetal hemoglobin (HbF), the individual is not suitable for treating with the modified TR cells.

(canceled)

(canceled)