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Breakthrough in gene therapy-based eye treatment

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Researchers have developed a method of delivering vision-restoring gene therapy, providing a new therapeutic option for patients with ocular disorders such as macular degeneration (AMD) and inherited retinal disorders.

This new technological advance was made by Nanoscope Technologies, supported by US body the National Institutes of Health (NIH).

The standard gene therapy approach uses viral vectors, which cannot be localised to damaged regions as it occurs in dry-AMD.

Further, viral vector has limitation in terms of gene-payload that it can carry.

Several therapeutic genes are large in size and currently there is limited option for their delivery.

While Nanoscope is using viral vector to deliver its proprietary ambient light-activatable Multi-Characteristic Opsin (MCO) for Advanced Retinitis Pigmentosa, the team is pioneering laser based spatially targeted gene delivery to partially degenerated retina such as geographic atrophies in dry-AMD.

Earlier, Nanoscope team has reported use of nanoparticles in combination with near-infrared laser for targeted gene delivery.

“However, this is the first-time laser delivery of opsin-genes has been achieved in-vivo without use of nanoparticles,” said Samarendra Mohanty,  chief scientific officer at Nanoscope.

Though the paper reports vision restoration in mice after ultrafast laser based MCO-gene delivery to mice with retinal degeneration, the team has been making progress in ultrafast laser delivery of large therapeutic genes for different ocular disorders.

“We are also advancing development of image-guided ultrafast laser gene delivery platform for clinical translation,” said Subrata Batabyal, first author of the paper.

In addition to functional improvement of vision observed after ultrafast laser delivery of MCO-gene, Nanoscope team is assessing behavioral improvement.

“Our team’s effort is unique and audacious. We will achieve our goal of restoring vision in patients suffering from many devastating ocular disorders, for which there is no therapy yet” said  CEO Sulagna Bhattacharya.


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