We have been trying to correct the mutations in the WFS1gene that cause disease manifestations using a new technology called CRISPR-CAS. This is a new type of gene therapy. We could finally correct one mutation in iPS cells from one patient with Wolfram syndrome. It took a few months, but this is a great first step. We will keep on trying!
Thank you for reading this blog. I feel grateful. Have a wonderful day. Onward & upward, Fumi Urano
Image courtesy of NIH Image Library