Versatile retinal protection through non-viral, intravitreal delivery of SARM1 siRNA (NEI R21)
Amount
Not specified
Deadline✓ Verified
Aug 31, 2028
Funder
National Eye Institute
Eligible
open to all
About This Grant
Project Summary Retinal degenerative diseases encompass a wide range of conditions, including inherited retinal diseases and age-related macular degeneration. These diseases lead to vision loss and impact over 300 million people globally. Ultimately, this vision loss is caused by the death of the photoreceptor cells (PRs). Delivering neuroprotective therapeutics that aim to halt cell death of PRs, regardless of disease etiology, would combat vision loss across the spectrum of retinal diseases. Currently, lack of safe, efficient delivery vehicles suitable for repeated administration of neuroprotective therapeutics is a major barrier to developing therapies for retinal diseases. We have developed and combined two distinct technologies to propel forward protective retinal therapeutics: 1) an effective siRNA targeting a pro-degenerative enzyme, SARM1, that confers functional protection against PR degeneration; and 2) unique lipid nanoparticles (LNPs) that have best-in-class tropism for PRs
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