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Our cells are Full of unrealized potential. Almost every human cell contains the genetic information needed to become any other type of cell. For example, skin cells have the same genes as muscle cells or brain neurons, but in each type of cell, only some of these genes are turned on, while others remain silent. It’s a bit like cooking different meals with the same ingredients in the cabinet. If we understand the formula behind each type of cell, then in theory we can use this information to design each cell type in the human body.
This is Markcott’s goal.Kurt is the CEO and co-founder Bit Bio-A company headquartered in Cambridge, England, hopes to revolutionize clinical research and drug discovery by producing precisely designed batches of human cells. The basic scientific research of new drugs and new therapies usually starts with tests on mice or the most widely used human cell line: kidney cells and cervical cancer cells. This can be a problem because the cells that are being tested may be significantly different from the cells that the drug candidate should target in the body. A drug that works in mice may prove ineffective when tested in humans. “No mouse on this planet has ever suffered from Alzheimer’s disease, it just doesn’t exist,” Kurt said.But test potential Alzheimer’s friends Studying a human brain cell designed to show signs of Alzheimer’s disease can show more clearly whether the drug is likely to be successful.
“Each cell type has its own small program or zip code-the combination of transcription factors that define it,” Cot said. By inserting the correct procedures into stem cells, researchers can activate the genes encoding these transcription factors and transform the stem cells into specific types of mature cells. Unfortunately, biology has a way to fight back. Cells often silence these genes, preventing the production of transcription factors. Kotter’s solution—discovered as part of his research at Cambridge University—is to insert this program into a region of the genome that prevents gene silencing, which Kotter calls a “gene safe harbor.”
Bit.bio currently sells two different reprogramming cell lines: muscle cells and a specific type of brain neurons, but its plan is to create customized cell lines for the pharmaceutical industry and academic research. “What we are doing now with our industry partners is to create disease-related genetic modifications,” Kotter said. He likened this method to running software on a computer. By inserting the correct code bits into the cell’s genome, you can control how the cell behaves. “This means that we can now run programs and we can reprogram human cells,” Kotter said. Cell reprogramming technology can also go far beyond model cell lines and contribute to the development of new treatment methods, such as cell therapy.
In some cell therapies, the patient’s own immune cells are grown outside the body, then modified and reinserted to help fight the disease—a long and expensive process. A cell therapy used to treat young people with leukemia costs more than £280,000 (US$371,400) per patient. Ramy Ibrahim, chief medical officer of Bit.bio, said that the company’s technology can help reduce the cost of cell therapy and make it easier to manufacture immune cells on a large scale. “We can now edit a large number of correct cell types, and I think this will be transformative,” he said.
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