For the first time ever, a person’s life has been saved by gene editing.
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Layla’s doctors got permission to use an experimental form of gene therapy using genetically engineered immune cells from a donor. Within a month these cells had killed off all the cancerous cells in her bone marrow.
Acute lymphoblastic leukemia and other blood tumors in which B-cells become malignant are extremely well-suited to this approach. You can cook up a T-cell that will react against B-cell specific proteins, inject it, and it will sense all the B cells around it and grow up to large numbers and kill all the B-cells and B-cell derived tumor cells in the patient’s body. You can live without B-cells (with a hit to immune system strength) and they have some very cell-specific proteins. Going after B-cell malignancies with modified immune cells has been successfully done before.
I am loving the new twist though—rather than going through the process of extracting the patient’s own T-cells and modifying them, they took a T-cell line they already had and destroyed its ability to ever respond to anything but the targeted antigen, meaning that a tissue-compatibility mismatch was irrelevant because it would never go after any foreign things it encountered other than the one coded target. The cells were apparently also modded to be resistant to chemotherapy drugs. The same cell line could be used in multiple people—though I’m sure that if any of the patient’s own immune system remained at all the foreign T cells would eventually be killed off rather than becoming a permanent part of the immune system as sometimes happens when the cells come from the patient themselves.
Gene editing saves girl dying from leukaemia in world first
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Acute lymphoblastic leukemia and other blood tumors in which B-cells become malignant are extremely well-suited to this approach. You can cook up a T-cell that will react against B-cell specific proteins, inject it, and it will sense all the B cells around it and grow up to large numbers and kill all the B-cells and B-cell derived tumor cells in the patient’s body. You can live without B-cells (with a hit to immune system strength) and they have some very cell-specific proteins. Going after B-cell malignancies with modified immune cells has been successfully done before.
I am loving the new twist though—rather than going through the process of extracting the patient’s own T-cells and modifying them, they took a T-cell line they already had and destroyed its ability to ever respond to anything but the targeted antigen, meaning that a tissue-compatibility mismatch was irrelevant because it would never go after any foreign things it encountered other than the one coded target. The cells were apparently also modded to be resistant to chemotherapy drugs. The same cell line could be used in multiple people—though I’m sure that if any of the patient’s own immune system remained at all the foreign T cells would eventually be killed off rather than becoming a permanent part of the immune system as sometimes happens when the cells come from the patient themselves.