According to Cheng, a good model to study the disease does not exist as engineered mice without the gene either die before birth, or do not reproduce symptoms found in patients.
So using a conventional genetic engineering tool, the researchers tried for years-literally-to knock out PIG-A in adult stem cells, without success. They then turned to knocking out PIG-A in human embryonic stem cells. Without certain GPI proteins, cells may not function properly. Then the researchers took one more step to verify that their engineered embryonic stem cells behaved like normal stem cells.
Frequently Asked Questions (FAQs)
One of the earliest steps of embryonic stem cell differentiation in normal embryonic development is the development of the trophoblast, a layer of seed cells that later develops into the placenta. Trophoblast differentiation, according to Cheng, occurs when embryonic stem cells are exposed to BMP-4 protein, either naturally or in lab. To their surprise, however, when they treated their knockout cells with BMP-4, the cells did not become trophoblasts. Only when they added the PIG-A gene back into their cells did BMP-4 do its work and cause the cells to become trophoblasts, allowing the researchers to conclude that trophoblast differentiation depends on certain cell surface proteins to receive the BMP-4 signal.
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All cells in the body contain the same genetic material.
chapter and author info
The difference between cells therefore depends solely on which genes are "turned on". This new knowledge will be crucial for stem cell therapy and potentially treating people with cancer. These are malignant tumors that start in fetal cells in the brain and mainly affect children up to four years old.