Part 7: Rare Disease

Part 7: Rare Disease.

The final two maroon and white wedge-shaped exhibits focus on Rare Diseases. 

Begin on the exterior panel closest to the stairs. While common diseases like cardiovascular disease and diabetes involve numerous genetic variants, rare diseases are caused by a single variant. An individual disease is considered rare when there are less than 200,000 individual cases. However, rare disease is quite common, affecting one in every ten people. Scientists can identify the variant that causes the disease but then must determine how to correct it to potentially cure the disease. CRISPR gene editing offers exciting possibilities for a cure. As you move to the exterior panel by the window and then back inside, you can see how scientists are working to develop a CRISPR based therapy that removes the disease-causing misspelling from the genetic code. 

However, it is challenging to deliver CRISPR based therapeutics to the right cells and tissues in the body. Learn how scientists are using a variety of strategies to tackle this challenge. 

Take a moment to listen to the experts in the Point of View digital exhibit on the interior panel of the remaining exhibit structure, if you would like to know more about the potential and value of rare disease research.

When you are ready, make your way to the oval blue and white table.