Genome editing will save the life of a leukemia girl for the first time in the world

It is said that the latest technology called "human beings have stepped into the realm of God" that gene selection can be made with much higher precision than conventional gene recombination technology is "Genome editing"is. Genomic editing is rapidly spreading application to animals and plants, but the application to human beings has not yet been decided rules, the stage where careful discussion is repeated. However, a case in which treatment using this genome editing saved the life of a leukemia girl has been reported from the UK.

Gene editing saves girl dying from leukaemia in world first | New Scientist
https://www.newscientist.com/article/dn28454-gene-editing-saves-life-of-girl-dying-from-leukaemia-in-world-first/


Leila, a hospital in the UK, who was an infant three months oldAcute lymphocytic leukemiaIt was diagnosed as.

Acute lymphocytic leukemia is a kind of blood cancer and it is a disease that is common in children. In the bone marrow, various blood cells are produced from the cell which becomes the seed of the blood cell called "hematopoietic stem cell", but this occurs abnormally in the process of differentiation into lymphocytes, the cell stops growing and it is in the bone marrow It is acute lymphocytic leukemia that proliferate, occupy the bone marrow and normal blood cells are not made.

After the symptoms were discovered, Ms. Leila immediately wrote "Great Ormond Street Hospital(GOSH) "and received standard chemotherapy. This "standard chemotherapy" was a cure that bone marrow transplantation returned the immune system to normal. "In the case of children older than Layla, the success rate of bone marrow transplantation increases," said Sujith Samarasinghe, who worked as a specialist in leukemia at GOSH and is also Layla's doctor. However, in the case of infants shortly after birth like Reira, the cure rate seems to be only about 25%, unfortunately, despite performing bone marrow transplantation, Layla's leukemia is not cured It was.


GOSH will immediately challenge the second bone marrow transplant surgery despite failure to treat bone marrow transplant once. The second surgery was to transplant the immune cells obtained from the donor to Mr. Leila's bone marrow and to have the immune cells attack and remove the cancer cells. However, leukemia recurs again in 2 months after surgery, and the second treatment also fails. "Usually when you come this far, it is a desperate situation," Paul Veys, the head of the GOSH bone marrow transplant surgery team. "But we could not give up on our daughter's life, so I grabbed the doctor to try anything," Lisa, Mr. Leila's mother.

Therefore, the bone marrow transplant surgery team is conducting gene therapy research at University College LondonWaseem Qasim's Research GroupI will inquire as to whether there is any treatment by mail.

If one of immune cells "T cellsIf it is injected into another patient and not completely harmonized, the T cell will attack the cell inside the patient's body as an enemy. If it is possible to suppress even this reaction, T cell can be a wonderful means of rescuing a patient who has abnormality in the immune system such as leukemia, so Qasim uses genetic manipulation technique called "genome editing" I was conducting research to delete only the "gene that recognizes other cells as enemies" of T cells received from the donor.

In conventional genetic recombination technology, we could only add genes to DNA, but "genome editing" has made it possible to manipulate genes with higher precision. If you read the following article you can tell what kind of technology "genome editing" is.

What technology is "genome editing" in God's domain that can freely design genes and change the characteristics of living things? - GIGAZINE


When the Qasim team got in touch from the bone marrow transplant team of GOSH, Qasim is the AmericanCellectisIt was said that it was at the stage of doing a mouse experiment of genome-edited T cell "UCART 19" developed in cooperation. "I was worried because the treatment with UCART 19 was not yet done for humans but we wanted you to try that treatment and I did not have any question because Layla It is because I was suffering from sickness so much and I had a need to do something, "Ashley, Leila's father says.

Following these circumstances, Ms. Leila underwent surgery to transplant the T cell "UCART 19" produced by genome editing into the bone marrow. This surgery seems to be the second case where genomic edited cells were used, and in the first case it was transplanted genome-edited T cells into HIV patients.

It was confirmed that within 1 month after surgery, T cells removed all cancer cells from Leila's bone marrow. After 3 months after surgery, a second bone marrow transplant is performed to restore the immune system. In this transplantation, Heira introduced healthy immune cells in the body of Layla and removed UCART 19 that entered the body by the first surgery. So, there are no genetically modified cells in the body of Layla at the moment. However, it is a case that cancer cells are not detected even after 1 or 2 years from surgery, so it will be possible to declare that it has cured completely, so that the inspection will be repeated periodically until then.

Although it is still premature to say "Successful treatment", Layla seems to be recovering smoothly.


Cellectis will start clinical trials of treatment using UCART 19 from 2016. Qasim is scheduled to be held in Florida in December 2015American Society of HematologyWe plan to announce the results of our research.