Blog 8 – Basics Behind Gene Therapy – My Sickle Cell Research Xplained


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Blog 8 – Basics Behind Gene Therapy

You might’ve heard the words “gene therapy” research for sickle cell disease being mentioned a lot in the news lately. Are you wondering what it’s all about? Let’s get into it.

What’s a gene?

Genes make us who we are—the color of our hair, how tall we are, our red blood cells, etc.

Genes are made up of DNA, which is the “recipe” for how to make something in our bodies. We get this “recipe” from each of our parents. What our bodies end up making is dependent on what’s written in the recipe; so for example, if you’re making a pizza, the type of pizza that you make depends on if your recipe calls for mozzarella or provolone cheese!

A Problem With the Recipe (the Gene)

In sickle cell disease, there’s a problem with the recipe for making hemoglobin, a substance used to carry oxygen in red blood cells. Without healthy hemoglobin, the red blood cells are deformed and stiff, which can lead to sickling or blockage in your blood vessels.

Gene Therapy

So what if you could fix the problem with the recipe? That’s gene therapy! A therapy to fix the problem at the source. This therapy is being researched for patients with sickle cell disease by targeting the root cause.

How does the healthy gene get into your cells? Think of the healthy gene as a recipe written in a letter. It needs to be delivered in an envelope. In this case, the “envelope” is from a virus that can’t cause disease, but carries the new recipe to your cells. The virus is only the packaging on the outside. The harmful parts of the virus are all removed so that the new recipe can be delivered safely. You want a good envelope that you know the cell will take in. For instance, if a UPS or FedEx driver were to give you a package, wouldn’t you be more likely to accept it than from a random person on the street? The virus gives the chance to deliver the recipe in the right packaging to get into the cells.

Gene Editing

On the other hand, gene editing is a process that is like using scissors to cut out a part of the recipe and then “glue” in a new recipe. Gene editing is being researched in sickle cell disease.

Until next time...

If you want to learn more, stay tuned for a future podcast featuring a sickle cell expert who will be discussing gene therapy a bit further! Remember, any therapy options should be discussed with your sickle cell provider and there are risks and benefits to every treatment plan.

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