biostudies-literatureOzawa YGrants-in-Aid for Scientific Research by the Japan Society of the Promotion of Science1497https://www.ebi.ac.uk/biostudies/studies/S-EPMC9099608biostudies-literatureUnknown11(9)Retinitis pigmentosa (RP) is a hereditary blinding disease characterized by gradual photoreceptor death, which lacks a definitive treatment. Here, we demonstrated the effect of 4-phenylbutyric acid (PBA), a chemical chaperon that can suppress endoplasmic reticulum (ER) stress, in P23H mutant rhodopsin knock-in RP models. In the RP models, constant PBA treatment led to the retention of a greater number of photoreceptors, preserving the inner segment (IS), a mitochondrial- and ER-rich part of the photoreceptors. Electroretinography showed that PBA treatment preserved photoreceptor function. At the early point, ER-associated degradation markers, <i>xbp1s</i>, <i>vcp</i>, and <i>derl1</i>, mitochondrial kinetic-related markers, <i>fis1</i>, <i>lc3</i>, and <i>mfn1</i> and <i>mfn2</i>, as well CellsEffects of Epigenetic Modification of PGC-1α by a Chemical Chaperon on Mitochondria Biogenesis and Visual Function in Retinitis Pigmentosa.PMC909960821K09683Ozawa YOkano HHomma KTsubota KOsada HToda ENagai NfalseEffects of Epigenetic Modification of PGC-1α by a Chemical Chaperon on Mitochondria Biogenesis and Visual Function in Retinitis Pigmentosa.Retinitis pigmentosa (RP) is a hereditary blinding disease characterized by gradual photoreceptor death, which lacks a definitive treatment. Here, we demonstrated the effect of 4-phenylbutyric acid (PBA), a chemical chaperon that can suppress endoplasmic reticulum (ER) stress, in P23H mutant rhodopsin knock-in RP models. In the RP models, constant PBA treatment led to the retention of a greater number of photoreceptors, preserving the inner segment (IS), a mitochondrial- and ER-rich part of the photoreceptors. Electroretinography showed that PBA treatment preserved photoreceptor function. At the early point, ER-associated degradation markers, <i>xbp1s</i>, <i>vcp</i>, and <i>derl1</i>, mitochondrial kinetic-related markers, <i>fis1</i>, <i>lc3</i>, and <i>mfn1</i> and <i>mfn2</i>, as well 2022-01-01T00:00:00Z2022 Apr2025-04-25T21:32:54.17Z2025-02-19T00:55:28.329ZS-EPMC90996083556380310.3390/cells11091497