TY - JOUR
T1 - Current Landscape of Treatments for Wolfram Syndrome
AU - Abreu, Damien
AU - Urano, Fumihiko
N1 - Publisher Copyright:
© 2019 The Author(s)
PY - 2019/10
Y1 - 2019/10
N2 - Wolfram syndrome is a rare genetic spectrum disorder characterized by insulin-dependent diabetes mellitus, optic nerve atrophy, and progressive neurodegeneration, and ranges from mild to severe clinical symptoms. There is currently no treatment to delay, halt, or reverse the progression of Wolfram syndrome, raising the urgency for innovative therapeutics for this disease. Here, we summarize our vision for developing novel treatment strategies and achieving a cure for Wolfram-syndrome-spectrum disorder.
AB - Wolfram syndrome is a rare genetic spectrum disorder characterized by insulin-dependent diabetes mellitus, optic nerve atrophy, and progressive neurodegeneration, and ranges from mild to severe clinical symptoms. There is currently no treatment to delay, halt, or reverse the progression of Wolfram syndrome, raising the urgency for innovative therapeutics for this disease. Here, we summarize our vision for developing novel treatment strategies and achieving a cure for Wolfram-syndrome-spectrum disorder.
KW - WFS1
KW - Wolfram syndrome
KW - endoplasmic reticulum
KW - gene therapy
KW - regenerative therapy
UR - http://www.scopus.com/inward/record.url?scp=85070489460&partnerID=8YFLogxK
U2 - 10.1016/j.tips.2019.07.011
DO - 10.1016/j.tips.2019.07.011
M3 - Short survey
C2 - 31420094
AN - SCOPUS:85070489460
SN - 0165-6147
VL - 40
SP - 711
EP - 714
JO - Trends in Pharmacological Sciences
JF - Trends in Pharmacological Sciences
IS - 10
ER -