Small Molecule Pharmacochaperones: Developing Oral Therapies to...

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2025-12-05 12:19:26 -

Small Molecule Pharmacochaperones: Developing Oral Therapies to Stabilize Misfolded Proteins in Retinitis Pigmentosa

A significant number of Retinitis Pigmentosa cases are caused by mutations, particularly in the Rhodopsin gene, that result in the production of a misfolded, toxic protein. This defective protein accumulates in the photoreceptor cells, leading to cellular stress, dysfunction, and eventual cell death. Rather than attempting to correct the gene, small molecule pharmacochaperones are being...

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