So what is gene therapy? A simple answer is that it's therapy for a
genetic disease, which begs the next question, what is a genetic disease?
Well, a gene is generally a piece of DNA that can be expressed to make a
protein, which then goes on to do something. Genes can mutate, which can
have many different effects on proteins. For tonight's talk, though,
we're going to be looking at simple mutations, which completely prevent
expression of the protein. What are the consequences of your body lacking
a protein?
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