Evidence map›Paper›PMID 40349106›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2025

Restoring AIBP expression in the retina provides neuroprotection in glaucoma.

Won-Kyu Ju, Keun-Young Kim, Tonking Bastola, Ziyao Shen, Seunghwan Choi, Guy A Perkins, Sinwoo Hwang, Jungsu Kim, Jin-Woo Kwon, Muna Poudel and 10 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

10 citing papers in PubMed.

  1. Review
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  7. Restoring AIBP expression in the retina provides neuroprotection in glaucoma.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

20 authors.

Won-Kyu JuHamilton Glaucoma Center, and Viterbi Family Department of Ophthalmology and Shiley Eye Institute, University of California, San Diego, La Jolla, CA 92039, USA. Electronic address: wju@health.ucsd.edu.
Keun-Young KimNational Center for Microscopy and Imaging Research, Department of Neurosciences, University of California, San Diego, La Jolla, CA 92039, USA.
Tonking BastolaHamilton Glaucoma Center, and Viterbi Family Department of Ophthalmology and Shiley Eye Institute, University of California, San Diego, La Jolla, CA 92039, USA.
Ziyao ShenHamilton Glaucoma Center, and Viterbi Family Department of Ophthalmology and Shiley Eye Institute, University of California, San Diego, La Jolla, CA 92039, USA.
Seunghwan ChoiHamilton Glaucoma Center, and Viterbi Family Department of Ophthalmology and Shiley Eye Institute, University of California, San Diego, La Jolla, CA 92039, USA.
Guy A PerkinsNational Center for Microscopy and Imaging Research, Department of Neurosciences, University of California, San Diego, La Jolla, CA 92039, USA.
Sinwoo HwangHamilton Glaucoma Center, and Viterbi Family Department of Ophthalmology and Shiley Eye Institute, University of California, San Diego, La Jolla, CA 92039, USA.
Jungsu KimDepartment of Medicine, University of California, San Diego, La Jolla, CA 92039, USA.
Jin-Woo KwonHamilton Glaucoma Center, and Viterbi Family Department of Ophthalmology and Shiley Eye Institute, University of California, San Diego, La Jolla, CA 92039, USA; Department of Ophthalmology and Visual Science, St. Vincent's Hospital, The Catholic University of Korea, Jungbu-daero 93, Paldal-gu, Suwon, Gyeonggi-do 16247, Republic of Korea.
Muna PoudelHamilton Glaucoma Center, and Viterbi Family Department of Ophthalmology and Shiley Eye Institute, University of California, San Diego, La Jolla, CA 92039, USA.
Sébastien PhanNational Center for Microscopy and Imaging Research, Department of Neurosciences, University of California, San Diego, La Jolla, CA 92039, USA.
Fan XiaDepartment of Ophthalmology and Visual Sciences, University of Texas Medical Branch, Galveston, TX 77555-0144, USA.
Shuizhen ShiDepartment of Ophthalmology and Visual Sciences, University of Texas Medical Branch, Galveston, TX 77555-0144, USA.
Hyunkyung ChoHamilton Glaucoma Center, and Viterbi Family Department of Ophthalmology and Shiley Eye Institute, University of California, San Diego, La Jolla, CA 92039, USA; Department of Ophthalmology, Gyeongsang National University Changwon Hospital, Gyeongsang National University, School of Medicine, Changwon, Gyeongsangnam-do 51472, Republic of Korea.
Hua LiuDepartment of Ophthalmology and Visual Sciences, University of Texas Medical Branch, Galveston, TX 77555-0144, USA.
Wenbo ZhangDepartment of Ophthalmology and Visual Sciences, University of Texas Medical Branch, Galveston, TX 77555-0144, USA.
Robert N WeinrebHamilton Glaucoma Center, and Viterbi Family Department of Ophthalmology and Shiley Eye Institute, University of California, San Diego, La Jolla, CA 92039, USA.
Mark H EllismanNational Center for Microscopy and Imaging Research, Department of Neurosciences, University of California, San Diego, La Jolla, CA 92039, USA.
Yury I MillerDepartment of Medicine, University of California, San Diego, La Jolla, CA 92039, USA; Raft Pharmaceuticals LLC, San Diego, CA 92121, USA.
Soo-Ho ChoiDepartment of Medicine, University of California, San Diego, La Jolla, CA 92039, USA. Electronic address: soc002@health.ucsd.edu.

Funding

Vision BiostatisticsP30EY022589 · NEI · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI Radha Ayyagari · 2012 to 2026
$10.3M
National Center for Microscopy and Imaging Research: A BRAIN Technology Integration and Dissemination ResourceU24NS120055 · NINDS · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI ELLISMAN, MARK H · 2021 to 2025
$5.4M
Mechanisms of Retinal Neuronal InjuryR01EY022694 · NEI · UNIVERSITY OF TEXAS MED BR GALVESTON · PI ZHANG, WENBO · 2012 to 2021
$3.8M
Reversing Microglial Inflammarafts and Mitochondrial Dysfunction in Alzheimer's DiseaseR01AG081037 · NIA · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI Mark H Ellisman, WONKYU JU · 2023 to 2026
$3.1M
The National Center for Microscopy and Imaging Research, a Community-wide Scientific ResourceR24GM137200 · NIGMS · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI ELLISMAN, MARK H · 2020 to 2022
$3.0M
Neuroprotective Role of Sirt6 in GlaucomaR01EY031054 · NEI · UNIVERSITY OF TEXAS MED BR GALVESTON · PI LIU, HUA · 2020 to 2024
$2.8M
Mitochondrial Protection in Glaucomatous Optic NeuropathyR01EY031697 · NEI · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI JU, WONKYU, PERKINS, GUY A · 2020 to 2023
$2.7M
AIBP-mediated neuroprotection in glaucomatous optic neuropathyR01EY034116 · NEI · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI SOO-HO CHOI, WONKYU JU · 2023 to 2026
$2.6M
Macrophage Inflammarafts and Mitochondrial Dysfunction in AtherosclerosisR01HL171505 · NHLBI · UNIVERSITY OF CALIFORNIA, SAN DIEGO · PI Yury Miller · 2024 to 2026
$2.3M
CXCR3-Mediated Cell-Cell Communication in GlaucomaR01EY034376 · NEI · UNIVERSITY OF TEXAS MED BR GALVESTON · PI Gracie Vargas, Fan Xia · 2022 to 2026
$2.2M
AAV-AIBP Therapy for Alzheimer's DiseaseR41AG081004 · NIA · RAFT PHARMACEUTICALS, LLC · PI JU, WONKYU, KOGAN, YAKOV · 2022 to 2023
$500k
NEI NIH HHS P30 EY022589NEI NIH HHS R01 EY022694NEI NIH HHS R01 EY031054NEI NIH HHS R01 EY031697NEI NIH HHS R01 EY034116NEI NIH HHS R01 EY034376NHLBI NIH HHS R01 HL171505NIA NIH HHS R01 AG081037NIA NIH HHS R41 AG081004NIGMS NIH HHS R24 GM137200NINDS NIH HHS U24 NS120055
6 · The paper itself

Abstract

Glaucoma is a neurodegenerative disease manifested by retinal ganglion cell (RGC) death and irreversible blindness. We have identified apolipoprotein A-I binding protein (AIBP) that controls excessive cholesterol accumulation and neuroinflammation in the retina by upregulating the cholesterol transporter ABCA1 and reducing TLR4 signaling and mitochondrial dysfunction. Here, we demonstrated that AIBP and ABCA1 expression were decreased, whereas Toll-like receptor 4 (TLR4), interleukin-1β (IL-1β), and the cholesterol content increased in the retina of patients with glaucoma and mouse models of glaucoma. Restoring AIBP deficiency by a single intravitreal injection of adeno-associated virus (AAV) protected RGCs and ameliorated visual dysfunction in experimental glaucoma. Conversely, AAV-mediated RGC-specific AIBP knockdown exacerbated RGC loss and visual dysfunction in a mouse model of glaucoma. Mechanistically, AAV-AIBP attenuated TLR4 and IL-1β expression and localization of TLR4 to lipid rafts, reduced cholesterol accumulation, and ameliorated visual dysfunction. Additionally, AAV-AIBP promoted mitochondrial complexity and function in Müller glia in vivo. Recombinant AIBP protein inhibited TLR4 and IL-1β activation and alleviated mitochondrial dysfunction in Müller glia in response to elevated pressure in vitro. These studies indicate that restoring AIBP expression in the glaucomatous retina reduces neuroinflammation and protects RGCs and Müller glia, suggesting the therapeutic potential of AAV-AIBP in human glaucoma.

Indexed as

GlaucomaNeuroprotectionRetinaAnimalsATP Binding Cassette Transporter 1CholesterolDependovirusDisease Models, AnimalEpendymoglial CellsGenetic TherapyGenetic VectorsHumansInterleukin-1betaMiceMitochondriaRetinal Ganglion CellsATP Binding Cassette Transporter 1CholesterolInterleukin-1betaToll-Like Receptor 4AAVAIBPcholesterolglaucomamitochondriaMüller glianeuroinflammationneuroprotectionRGCTLR4

Identifiers

PMID40349106
PMCPMC12461634

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.