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Posted On: 06/29/2020 9:42:59 PM
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Dr. Urano June 2020 Update
...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 retro-orbital (for brain)
injections....
...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 retro-orbital (for brain)
injections....
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