bioRxivJClub
Identification of Kinome Biomarkers and Therapeutic Rescue in cGMP-Linked Retinal Degeneration
bioRxiv · · Preprint · Open access
Topcu Okan, Ahmadian + 13 more
Abstract ↗AI summary
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Kinome analysis in peripheral blood mononuclear cells distinguishes healthy versus diseased states and captures therapy-associated signatures in rd10 mice treated with LP-CN03.
- Why it matters: Inherited retinal degenerations like RP and LCA have many causative genes, leaving limited systemic biomarkers for tracking progression or treatment response.
- What they did: The study performed kinome activity profiling in peripheral blood mononuclear cells and evaluated LP-CN03 in rd10 mice and in CRISPR-Cas9-generated Aipl1-/- mice using retinal imaging, electroretinography, optomotor testing, and photoreceptor gene expression.
- The result: LP-CN03 preserved retinal structure and function in Aipl1-/- mice, supporting a neuroprotective effect in cGMP-related RP and LCA and positioning kinome profiling as a translational readout.
The findingWhy it mattersWhat they didThe result
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