Retinal prolactin isoform PRLΔE1 sustains rod disease in inherited retinal degenerations

Abstract PRLΔE1, a retina-specific isoform of prolactin, is expressed in multiple and diverse forms of canine inherited retinal degeneration (IRD). We find that while PRLΔE1 expression in rods is not associated with the initial phase of disease characterized by acute photoreceptor cell death, it is...

Full description

Saved in:
Bibliographic Details
Main Authors: Raghavi Sudharsan, Jennifer Kwok, Malgorzata Swider, Alexander Sumaroka, Gustavo D. Aguirre, Artur V. Cideciyan, William A. Beltran
Format: Article
Language:English
Published: Nature Publishing Group 2024-09-01
Series:Cell Death and Disease
Online Access:https://doi.org/10.1038/s41419-024-07070-1
Tags: Add Tag
No Tags, Be the first to tag this record!
_version_ 1849333969342955520
author Raghavi Sudharsan
Jennifer Kwok
Malgorzata Swider
Alexander Sumaroka
Gustavo D. Aguirre
Artur V. Cideciyan
William A. Beltran
author_facet Raghavi Sudharsan
Jennifer Kwok
Malgorzata Swider
Alexander Sumaroka
Gustavo D. Aguirre
Artur V. Cideciyan
William A. Beltran
author_sort Raghavi Sudharsan
collection DOAJ
description Abstract PRLΔE1, a retina-specific isoform of prolactin, is expressed in multiple and diverse forms of canine inherited retinal degeneration (IRD). We find that while PRLΔE1 expression in rods is not associated with the initial phase of disease characterized by acute photoreceptor cell death, it is associated with the protracted phase of slow cell loss. Restoration of photoreceptors to a healthy state by gene-specific replacement therapy of individual IRDs successfully suppresses PRLΔE1 expression. Moreover, short-term PRLΔE1 silencing using shRNA results in preservation of outer nuclear layer thickness, suggesting PRLΔE1 drives retinal disease. However, longer-term observations reveal off-target toxic effects of the PRLΔE1 shRNA, precluding determination of its full therapeutic potential. Future research efforts aimed at enhancing the safety and specificity of PRLΔE1-targeting strategies may identify a potential universal intervention strategy for sustaining photoreceptors during the prolonged phase of multiple IRDs.
format Article
id doaj-art-e37984bba17c4ae580d9b5b917c7b0d9
institution Kabale University
issn 2041-4889
language English
publishDate 2024-09-01
publisher Nature Publishing Group
record_format Article
series Cell Death and Disease
spelling doaj-art-e37984bba17c4ae580d9b5b917c7b0d92025-08-20T03:45:41ZengNature Publishing GroupCell Death and Disease2041-48892024-09-0115911010.1038/s41419-024-07070-1Retinal prolactin isoform PRLΔE1 sustains rod disease in inherited retinal degenerationsRaghavi Sudharsan0Jennifer Kwok1Malgorzata Swider2Alexander Sumaroka3Gustavo D. Aguirre4Artur V. Cideciyan5William A. Beltran6Division of Experimental Retinal Therapies, Department of Clinical Sciences & Advanced Medicine, School of Veterinary Medicine, University of PennsylvaniaDivision of Experimental Retinal Therapies, Department of Clinical Sciences & Advanced Medicine, School of Veterinary Medicine, University of PennsylvaniaCenter for Hereditary Retinal Degenerations, Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania Perelman School of MedicineCenter for Hereditary Retinal Degenerations, Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania Perelman School of MedicineDivision of Experimental Retinal Therapies, Department of Clinical Sciences & Advanced Medicine, School of Veterinary Medicine, University of PennsylvaniaCenter for Hereditary Retinal Degenerations, Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania Perelman School of MedicineDivision of Experimental Retinal Therapies, Department of Clinical Sciences & Advanced Medicine, School of Veterinary Medicine, University of PennsylvaniaAbstract PRLΔE1, a retina-specific isoform of prolactin, is expressed in multiple and diverse forms of canine inherited retinal degeneration (IRD). We find that while PRLΔE1 expression in rods is not associated with the initial phase of disease characterized by acute photoreceptor cell death, it is associated with the protracted phase of slow cell loss. Restoration of photoreceptors to a healthy state by gene-specific replacement therapy of individual IRDs successfully suppresses PRLΔE1 expression. Moreover, short-term PRLΔE1 silencing using shRNA results in preservation of outer nuclear layer thickness, suggesting PRLΔE1 drives retinal disease. However, longer-term observations reveal off-target toxic effects of the PRLΔE1 shRNA, precluding determination of its full therapeutic potential. Future research efforts aimed at enhancing the safety and specificity of PRLΔE1-targeting strategies may identify a potential universal intervention strategy for sustaining photoreceptors during the prolonged phase of multiple IRDs.https://doi.org/10.1038/s41419-024-07070-1
spellingShingle Raghavi Sudharsan
Jennifer Kwok
Malgorzata Swider
Alexander Sumaroka
Gustavo D. Aguirre
Artur V. Cideciyan
William A. Beltran
Retinal prolactin isoform PRLΔE1 sustains rod disease in inherited retinal degenerations
Cell Death and Disease
title Retinal prolactin isoform PRLΔE1 sustains rod disease in inherited retinal degenerations
title_full Retinal prolactin isoform PRLΔE1 sustains rod disease in inherited retinal degenerations
title_fullStr Retinal prolactin isoform PRLΔE1 sustains rod disease in inherited retinal degenerations
title_full_unstemmed Retinal prolactin isoform PRLΔE1 sustains rod disease in inherited retinal degenerations
title_short Retinal prolactin isoform PRLΔE1 sustains rod disease in inherited retinal degenerations
title_sort retinal prolactin isoform prlδe1 sustains rod disease in inherited retinal degenerations
url https://doi.org/10.1038/s41419-024-07070-1
work_keys_str_mv AT raghavisudharsan retinalprolactinisoformprlde1sustainsroddiseaseininheritedretinaldegenerations
AT jenniferkwok retinalprolactinisoformprlde1sustainsroddiseaseininheritedretinaldegenerations
AT malgorzataswider retinalprolactinisoformprlde1sustainsroddiseaseininheritedretinaldegenerations
AT alexandersumaroka retinalprolactinisoformprlde1sustainsroddiseaseininheritedretinaldegenerations
AT gustavodaguirre retinalprolactinisoformprlde1sustainsroddiseaseininheritedretinaldegenerations
AT arturvcideciyan retinalprolactinisoformprlde1sustainsroddiseaseininheritedretinaldegenerations
AT williamabeltran retinalprolactinisoformprlde1sustainsroddiseaseininheritedretinaldegenerations