Question

Your classmate wants to talk to you about goats. It turns out that there is a company that has made genetically engineered goats that make the protein antithrombin in their milk. Normally, antithrombin in only made in the liver, and it goes into the blood and helps prevent blood clots. Some people can’t make antithrombin, so they are prone to large, sometimes deadly clots. The company collects the antithrombin from their goat’s milk and sells it as a drug, so that these patients can safely survive bloody procedures like surgeries and childbirth.

But your classmate is concerned about the goats. “All the cells of these goats have the company’s DNA for making the extra antithrombin. Think about all that extra antithrombin in their bodies- are they going to be able to make blood clots at all? It’s not right to create these sick animals.”

You say, “It says that the goats only make the extra antithrombin in their milk.”

Your classmate says, “I don’t believe that. How is it even possible to make the goats only make the antithrombin in their milk?”

Using the language of BILD 1, including promoter, enhancer, and transcription factor, explain to your classmate:

- How a piece of DNA could direct the protein to only be made in the milk, and
- What is different about the cells of the milk glands that causes antithrombin to be

made there and not in the goat’s other cells?

Answer 1

Answers:

1) DNA segment contains a mammary-specific promoter sequence that codes for the protein of interest and the regulatory DNA that tells the cell how to manufacture the protein.

2) ATryn is indicated for the prevention of peri-operative and peripartum thromboembolic events in patients with hereditary antithrombin deficiency. HD is a genetic disorder leading to excessive clotting because of a lack of normal antithrombin activity. Antithrombin (AT) is a 432 amino acid protein that regulates thrombin and the downstream clotting processes. AT becomes the treatment of choice as a prophylactic agent during periods of high risk of thrombosis. There are two forms of AT available — the plasma products taken from pooled whole blood and the new ATryn, transgenic recombinant antithrombin. The amino acid sequences of ATryn and human antithrombin are indistinguishable.Biopharmaceuticals has found that the intracellular machinery needed to properly fold and assemble the heavy and light chains of antibodies are functional within these cells.

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