To Study the potential for Collagen Mimetic Peptides to reduce progressive damage to the Bruch's-RPE-Photoreceptor Complex in Choroideremia.

Awardee: Malia Edwards

Institution: Johns Hopkins University

Grant Amount: $64,735.00

Funding Period: February 1, 2024 - January 31, 2025


Summary:

Collagen is the most common protein in the human. It is found in all organs in either its structural form or as a major component of the extracellular matrix. The source of newly formed collagen arising during tissue growth and remodeling is the nearby cells, often mesenchymal but also epithelial. A healthy body undergoes collagen remodeling on a constant basis, but aging or gene deficiencies can cause the ability to produce replacement collagen to decrease. In certain disease states collagen-producing cells are often involved and may become damaged as well, leading to a reduction in collagen replacement which slows the tissue healing process. A therapeutic with the ability to repair damaged collagen can facilitate recovery and halt progressive damage to critical tissues and organs, like the eye, when it is subjected to such diseases. CMPs are vastly different from denatured collagen supplements, which are highly advertised nutraceutical products and which do not have the direct collagen repairative activities associated with CMPs. CMPs have been shown to be efficacious and safe in humans when applied topically as eyedrops and systemically in animals under FDA regulated toxicity studies.

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CSF dynamics in mucopolysaccharidosis type I

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Leveraging predictive models to design high-throughput assays to resolve variants of uncertain significance (VUS) in SYNGAP1