COMPOSITIONS AND METHODS TO TREATING HEMOGLOBINOPATHIES
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Abstract
Embodiments herein provide specially designed synthetic BCL11A-targeting microRNAs for RNA polymerase II expression, and methods of use to treat hemoglobinopathies such as sickle cell disease or thalassemia by increasing the expression levels of fetal hemoglobin levels.
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Citations
134 Claims
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1-117. -117. (canceled)
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118. A hematopoietic stem or progenitor cell comprising a lentiviral vector that comprises a nucleic acid encoding:
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a) a first BCL11A segment that comprises a BCL11A sequence, a loop segment; and b) a second BCL11A segment arranged in tandem in a 5′
to 3′
direction,wherein the loop segment is between and directly linked to the first and second BCL11A segments, wherein the second BCL11A segment is complementary to the first BCL11A segment so that the first and second BCL11A segments base pair to form a hairpin loop with the loop segment forming the loop portion of the hairpin loop thus formed; and wherein the first BCL11A segment starts with a -GCGC- at the 5′
end and the second BCL11A segment ends with a -GCGC- at the 3′
end. - View Dependent Claims (119, 120, 121, 122, 123, 124, 125, 126, 127, 128, 129, 130, 131, 132, 133, 134)
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Specification