Base Editing Gene Therapy- Dr. Fumihiko Urano, MD, PhD

In collaboration with Dr. David Liu’s team at Harvard University/Broad Institute and Dr. Catherine Verfaillie’s team at the Katholieke Universiteit Leuven, we have been developing a novel gene therapy called Base Editing for Wolfram syndrome. This technology uses some components from CRISPR systems together with other enzymes to directly replace abnormal WFS1 gene with normal WFS1 gene. Although we are still at the early preclinical stage using cell models of Wolfram, we hope that we can bring this technology to our patients in the next 3-10 years. Please stay tuned.
/by

Regenerative Gene Therapy- Dr. Fumihiko Urano, MD, PhD

My current focus is to develop gene therapy for Wolfram syndrome. Our ultimate goal is to provide a cure using regenerative gene therapy. We have been trying to improve diabetes, visual acuity, and brain functions using viral vectors of a healthy Wolfram gene and a regenerative factor called MANF in mouse models. We are getting encouraging preliminary results and have published two articles recently. We are currently testing two ways to deliver genes through intravitreal (for optic nerve) and intraventricular (for brain) injections.
/by

OCTA in neurodegenerative optic neuropathies: emerging biomarkers at the eye–brain interface

October 1, 2020:

The use of OCTA (Optical coherence tomography angiography) in finding biomarkers for neurodegenerative diseases.

https://journals.sagepub.com/doi/full/10.1177/2515841420950508

/by