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Restoring vision in mice with retinal degeneration using multicharacteristic opsin
Author(s): Weldon W. Wright; Sivakumar Gajjeraman; Subrata Batabyal; Sanjay Pradhan; Sulagna Bhattacharya; Vasu Mahapatra; Ashutosh Tripathy; Samarendra K. Mohanty
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Paper Abstract

Retinal degenerative diseases, such as retinitis pigmentosa (RP) and dry age-related macular degeneration, have led to loss of vision in millions of individuals. Currently, no surgical or medical treatment is available, although optogenetic therapies are in clinical development. We demonstrate vision restoration using multicharacteristics opsin (MCO1) in animal models with degenerated retina. MCO1 is reliably delivered to specific retinal cells via intravitreal injection of adeno-associated virus (vMCO1), leading to significant improvement in visually guided behavior conducted using a radial arm water maze. The time to reach the platform and the number of error arms decreased significantly after delivery of MCO1. Notably, the improvement in visually guided behavior was observed even at light intensity levels orders of magnitude lower than that required for channelrhodopsin-2 opsin. Viability of vMCO1-treated retina is not compromised by chronic light exposure. Safe virus-mediated MCO1 delivery has potential for effective gene therapy of diverse retinal degenerations in patients.

Paper Details

Date Published: 18 August 2017
PDF: 8 pages
Neurophoton. 4(4) 041505 doi: 10.1117/1.NPh.4.4.041505
Published in: Neurophotonics Volume 4, Issue 4
Show Author Affiliations
Weldon W. Wright, Nanoscope Technologies, LLC (United States)
Sivakumar Gajjeraman, Nanoscope Technologies, LLC (United States)
Subrata Batabyal, Nanoscope Technologies, LLC (United States)
Sanjay Pradhan, Nanoscope Technologies, LLC (United States)
Sulagna Bhattacharya, Nanoscope Technologies, LLC (United States)
Vasu Mahapatra, NanoScope Technologies, LLC (United States)
Ashutosh Tripathy, Nanoscope Technologies, LLC (United States)
Samarendra K. Mohanty, Nanoscope Technologies, LLC (United States)

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