piperidines has been researched along with Stargardt-Disease* in 2 studies
1 review(s) available for piperidines and Stargardt-Disease
| Article | Year |
|---|---|
Novel therapeutics for Stargardt disease.
Stargardt disease, an inherited macular dystrophy caused by mutations in the ABCA4 gene encoding a retinal transporter protein, is the most prevalent form of macular degeneration in children. Patients with Stargardt disease develop severe vision loss within their first or second decades of life, which progresses to irreversible decreased visual acuity in almost all cases. Presently, there are no standard treatments for Stargardt disease. However, encouraging progress has been made in the development of innovative approaches to preventing vision loss in Stargardt patients.. Among the promising treatment candidates include ALK-001, fenretinide, and A1120 as pharmacological agents to modulate the visual cycle, StarGen Topics: Antineoplastic Agents; Fenretinide; Genetic Therapy; Humans; Ligands; Macular Degeneration; Piperidines; Retinal Pigment Epithelium; Stargardt Disease; Stem Cell Transplantation | 2017 |
1 other study(ies) available for piperidines and Stargardt-Disease
| Article | Year |
|---|---|
Design, synthesis, and evaluation of nonretinoid retinol binding protein 4 antagonists for the potential treatment of atrophic age-related macular degeneration and Stargardt disease.
Accumulation of lipofuscin in the retina is associated with pathogenesis of atrophic age-related macular degeneration and Stargardt disease. Lipofuscin bisretinoids (exemplified by N-retinylidene-N-retinylethanolamine) seem to mediate lipofuscin toxicity. Synthesis of lipofuscin bisretinoids depends on the influx of retinol from serum to the retina. Compounds antagonizing the retinol-dependent interaction of retinol-binding protein 4 (RBP4) with transthyretin in the serum would reduce serum RBP4 and retinol and inhibit bisretinoid formation. We recently showed that A1120 (3), a potent carboxylic acid based RBP4 antagonist, can significantly reduce lipofuscin bisretinoid formation in the retinas of Abca4(-/-) mice. As part of the NIH Blueprint Neurotherapeutics Network project we undertook the in vitro exploration to identify novel conformationally flexible and constrained RBP4 antagonists with improved potency and metabolic stability. We also demonstrate that upon acute and chronic dosing in rats, 43, a potent cyclopentyl fused pyrrolidine antagonist, reduced circulating plasma RBP4 protein levels by approximately 60%. Topics: Animals; Atrophy; Chemistry Techniques, Synthetic; Drug Design; Ligands; Macular Degeneration; Male; Mice; Molecular Docking Simulation; Piperidines; Prealbumin; Protein Conformation; Rats; Retinol-Binding Proteins, Plasma; Stargardt Disease; Structure-Activity Relationship | 2014 |