Genetic engineering saved the first human leukemia
Genetically modified cells for the first time helped to save a terminally ill child from leukemia. It is reported by New Scientist.
At the age of three months, Leila was diagnosed with acute lymphoblastic leukemia - a malignant disease in which cancerous bone marrow stem cells released into the blood immature lymphoid cells in large quantities. The girl immediately began to treat with standard treatments (chemotherapy and bone marrow transplantation), but at her age, this therapy is successful only in a quarter of cases, and Leila had no luck.
Despite the presence of cancer cells after chemotherapy sessions, the doctors decided to transplant bone marrow, hoping that the immune cells in the donor organ will win leukemia - but that did not happen. Two months later, the condition of Leyla again deteriorated, while doctors have turned to Qasim Waseem (Waseem Qasim) from University College London, who is developing genetic techniques to fight cancer.
In general, treatment is taking immune cells from a patient, genetically modify them to combat cancer cells and return back. Sometimes, there are added receptor gene CAR19, which causes cytotoxic T lymphocytes (T cells) to seek and kill cells with the CD19 protein on the surface - they cause acute lymphoblastic leukemia.
However, Leila was too small and the patient, to provide such a T-cell. Therefore, Qasim had to use material obtained from another healthy donor and a modified way to treat hundreds of patients. To these cells did not take all the cells in the recipient as foreign and attacked them, it took a gene editing with "molecular scissors" - proteins TALEN.
Qasim cut two genes: receptor responsible for recognizing the recipient's cells as foreign, and another gene whose absence made the donor T cells invisible to the antibodies that Leila was adopted (as a drug that suppresses its own immune system).
At the time, the modified T cells UCART19 been tested only on mice, but because the girl died, her parents decided to take a chance. Despite the likelihood of incorrect operation of the molecular scissors (sometimes they are not cut out those genes, and donor cells themselves turn into cancer), treatment was successful: T-cell lymphoblasts successfully overcame.
Three months later, Leila again transplanted bone marrow: a healthy immune cells recognize cells UCART19 as foreign and destroy them. Thus, now in the body girl remained genetically modified cells.
While a complete cure Leila too early to say, but she is alive and doing well. Complete clinical trials UCART19 will begin in 2016, which may prove that the success of the London girls were not accidental.
Genetically modified cells for the first time helped to save a terminally ill child from leukemia. It is reported by New Scientist.
At the age of three months, Leila was diagnosed with acute lymphoblastic leukemia - a malignant disease in which cancerous bone marrow stem cells released into the blood immature lymphoid cells in large quantities. The girl immediately began to treat with standard treatments (chemotherapy and bone marrow transplantation), but at her age, this therapy is successful only in a quarter of cases, and Leila had no luck.
Despite the presence of cancer cells after chemotherapy sessions, the doctors decided to transplant bone marrow, hoping that the immune cells in the donor organ will win leukemia - but that did not happen. Two months later, the condition of Leyla again deteriorated, while doctors have turned to Qasim Waseem (Waseem Qasim) from University College London, who is developing genetic techniques to fight cancer.
In general, treatment is taking immune cells from a patient, genetically modify them to combat cancer cells and return back. Sometimes, there are added receptor gene CAR19, which causes cytotoxic T lymphocytes (T cells) to seek and kill cells with the CD19 protein on the surface - they cause acute lymphoblastic leukemia.
However, Leila was too small and the patient, to provide such a T-cell. Therefore, Qasim had to use material obtained from another healthy donor and a modified way to treat hundreds of patients. To these cells did not take all the cells in the recipient as foreign and attacked them, it took a gene editing with "molecular scissors" - proteins TALEN.
Qasim cut two genes: receptor responsible for recognizing the recipient's cells as foreign, and another gene whose absence made the donor T cells invisible to the antibodies that Leila was adopted (as a drug that suppresses its own immune system).
At the time, the modified T cells UCART19 been tested only on mice, but because the girl died, her parents decided to take a chance. Despite the likelihood of incorrect operation of the molecular scissors (sometimes they are not cut out those genes, and donor cells themselves turn into cancer), treatment was successful: T-cell lymphoblasts successfully overcame.
Three months later, Leila again transplanted bone marrow: a healthy immune cells recognize cells UCART19 as foreign and destroy them. Thus, now in the body girl remained genetically modified cells.
While a complete cure Leila too early to say, but she is alive and doing well. Complete clinical trials UCART19 will begin in 2016, which may prove that the success of the London girls were not accidental.
Комментариев нет:
Отправить комментарий