Britannica Sources used in this answer

How do genetically modified mammalian cells produce factor VIII for people with hemophilia?

Created from Britannica articles using AI. AI can make mistakes, so verify using Britannica articles.

Rows of genetically modified barley plants grow in an outdoor test plot in Germany, with a yellow marker labeled 3 and protective netting overhead. — genetically modified organism Genetically modified crops are assessed for their impact on environmental factors such as soil quality.

Genetically modified (GM) mammalian cells are used to produce factor VIII for individuals with hemophilia A by serving as biological factories for the protein.1

In this process, researchers introduce the gene responsible for encoding human factor VIII into the genome of mammalian cells. These modified cells are then grown in laboratory cultures under controlled conditions. Because these cells are mammalian, they are capable of synthesizing and secreting the complex protein in a form that is medically effective for human use.1

This method is a significant application of recombinant DNA technology, which allows for the reliable, large-scale production of therapeutic proteins. By utilizing mammalian cell lines, scientists can ensure that the resulting factor VIII is properly processed, providing a safer and more consistent treatment option for patients compared to traditional plasma-derived concentrates.21