Wolfram Syndrome: Diagnosis, Management, and Treat

Wolfram Syndrome: Diagnosis, Management, and Treatment
Fumihiko Urano

Regenerative Therapy and Gene Therapy for Wolfram
syndrome
Our first goal is to stop the progression of Wolfram syndrome
using FDA-approved drugs or novel small molecules
targeting the ER. In parallel, we need to figure out a
way to replace damaged tissues, such as pancreatic β cells
and retinal cells, in patients with Wolfram syndrome. Rapid
progress in the field of regenerative medicine may
make this possible in the near future [42] (Fig. 2). Our
strategy is to create induced pluripotent stem (iPS) cells
using patients’ skin cells, correct WFS1 gene mutations
with genome editing technology, and differentiate these
iPS cells into insulin-producing β cells, retinal cells, and
neurons for transplantation [43•]. We are currently using
the Clustered Regularly Interspaced Short Palindromic
Repeats (CRISPR) technology. We are also testing if mesencephalic
astrocyte-derived neurotrophic factor (MANF),
a regeneration factor purified from astrocytes, can prevent
cell death and activate the proliferation of remaining β
cells, neurons, and retinal ganglion cells by leveraging
our natural ability to regenerate damaged tissues

http://www.wolframsyndrome.co.uk/docs/2016_Ur...tesRep.pdf