https://wolframsyndrome.dom.wustl.edu/rare-disease
Post# of 30028
2. Complete preclinical studies for gene therapy for optic nerve atrophy in Wolfram.
We have established six new adeno-associated virus constructs for gene therapy. We can transfer and express normal WFS1 gene and MANF gene (regeneration factor) in human cells. We are still at the preclinical stage, and I want to accelerate the speed. I have a few strategies I plan to share with you soon.