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A cure for Wolfram syndrome could lead to a cure for diabetes and blindness.

Sunday, May 11, 2014

Patient-based therapeutics part 10: Molecular Surgery 1

We have three major strategies for developing novel treatments and providing a cure for Wolfram syndrome. These are:

1. Develop drugs that can stop or delay the progression of declining brain and eye functions, and diabetes.
2. Develop drugs that can restore the function of damaged brain cells and pancreatic beta cells.
3. Replace damaged tissues using induced pluripotent stem cells (iPS cells) derived from skin cells of Wolfram syndrome patients.

We will focus on the third strategy today. This strategy has one major challenge. Even if we can successfully make high-quality eye cells and brain cells from iPS cells of Wolfram syndrome patients, these cells may be still susceptible to cell death because of the mutations in the WFS1 gene. To overcome this challenge, we are conducting "molecular surgeries" in these cells.

Using an engineered enzyme and artificial DNA, we are replacing a "disease-causing DNA sequence" with a "healthy DNA sequence." We have designed a sequence of procedures and are moving forward step by step. We designed four artificial DNA sequences so far and could confirm that all of these properly worked in a test tube last week! So we will move on to the next step. I will keep you updated about our progress. I was excited by our progress last week, and appreciated my team's efforts.

Happy Mother's Day to All Mothers!