Engineered hematopoietic stem cells for the treatment of acute myeloid leukemia
AssigneesLoma Linda University, Loma Linda University Medical Center, Faculty Physicians and Surgeons of Loma Linda University School of Medicine
InventorsHuynh CAO, Yi XU, Kimberly PAYNE, David BAYLINK
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Abstract
In one aspect, engineered hematopoietic stem cells comprising a heterologous expression cassette are provided. In some embodiments, the expression cassette comprises a promoter operably linked to a polynucleotide that encodes a 1α-hydroxylase protein, wherein the 1α-hydroxylase protein is human cytochrome P450 family 27 subfamily B member 1 (CYP27B1).
Claims31
An engineered hematopoietic stem cell comprising a heterologous expression cassette, the expression cassette comprising a promoter operably linked to a polynucleotide that encodes a 1α-hydroxylase protein, wherein the 1α-hydroxylase protein is human cytochrome P450 family 27 subfamily B member 1 (CYP27B1).
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A method of treating a human subject having a leukemia, the method comprising:
- administering to the subject a population of engineered hematopoietic stem cells or a pharmaceutical composition comprising a population of engineered hematopoietic stem cells, wherein the engineered hematopoietic stem cells comprise a heterologous expression cassette that comprises a promoter operably linked to a polynucleotide that encodes a human CYP27B1 protein.
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