CRISPR/Cas9 allows scientists to find and adjust or replace genetic defects.
LONDON – British scientists have developed a genome editing tool known as CRISPR/Cas9 to knock-out gene in embryos only a few days old, the testing of the technique the ability to decipher key gene functions in the early development of the human being.
The researchers said that their experiments, the use of a technology that is the subject of fierce international debate because of the fear that it would be used for making babies to order, it will deepen our understanding of the biology of early human development.
CRISPR/Cas9 allows scientists to find and adjust or replace genetic defects. Many describe it as game-changing.
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“One way to find out what a gene is in the development of the embryo is to see what happens if it doesn’t work,” said Kathy Niakan, a stem cell scientists who led the study in the Uk the Francis Crick Institute.
“Now we have shown that an efficient way to do this, we hope that other scientists will use it to find out the roles of other genes.”
She said that her hope was for scientists to decipher the roles of all the important genes, the embryos have to develop with success. This can then improve IVF treatments for infertile couples and also help doctors understand why so many pregnancies fail.
“It may take years for the achievement of a good understanding, our study is only the first step,” Niakan said.
Niakan, the team decided to use it to stop a key-gen for the production of a protein called OCT4, which is normally active in the first few days of human embryo development.
She spent more than a year to optimise the performance of their various techniques using the mouse embryos and human embryonic stem cells in the laboratory dishes, for the start of the work on human embryos.
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To inactivate OCT4, they used CRISPR/Cas9 to change the DNA of 41 human embryos. After seven days, the embryo development was stopped and the embryos were analyzed.
After an egg is fertilized, divides up to about seven days, it forms a ball of about 200 cells called a blastocyst, Niakan explained in a briefing on her work.
Her results, published in the journal Nature on Wednesday, found that human embryos require OCT4 to a blastocyst. Without it, the blastocyst cannot form or develop normally.
The Uk team work comes on the heels of the milestone of sciences in the United States, where scientists said in July that they had succeeded in changing the genes of a human embryo to recover from a disease-causing mutation.
Rob Buckle, chief science officer at the british Medical Research Council, praised Niakan the research and findings:
“Genome editing technologies – in particular the CRISPR-Cas9 used in this study – having a game-changing effect on our ability to understand the function of important human genes,” he said.