ArticleInvestigative ophthalmology & visual science2014
AAV-mediated lysophosphatidylcholine acyltransferase 1 (Lpcat1) gene replacement therapy rescues retinal degeneration in rd11 mice.
Article in Investigative ophthalmology & visual science, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.
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Who cites it
18 citing papers in PubMed, 25 citations in OpenAlex.
- Lysophosphatidylcholine acyltransferase 1 promotes head and neck squamous cell carcinoma progression by enhancing COX17-dependent oxidative phosphorylation.Cell death discovery · 2026Article
- LPCAT1, the Enzyme Responsible for Converting LPC to PC, Promotes OPC Differentiation In Vitro.Journal of cellular and molecular medicine · 2025Article
- LPCAT1 promotes melanoma cell proliferation via Akt signaling.Oncology reports · 2024Article
- Lysophosphatidylcholine acyltransferase 1 controls mitochondrial reactive oxygen species generation and survival of retinal photoreceptor cells.The Journal of biological chemistry · 2022Article
- A novel lipid prodrug strategy for sustained delivery of hexadecyloxypropyl 9-[2-(phosphonomethoxy)ethyl]guanine (HDP-PMEG) on unwanted ocular proliferation.Drug delivery · 2017Article
- CLINICAL PROGRESS IN INHERITED RETINAL DEGENERATIONS: GENE THERAPY CLINICAL TRIALS AND ADVANCES IN GENETIC SEQUENCING.Retina (Philadelphia, Pa.) · 2017Review
- Long-term Characterization of Retinal Degeneration in Royal College of Surgeons Rats Using Spectral-Domain Optical Coherence Tomography.Investigative ophthalmology & visual science · 2017Article
- Long-term retinal cone rescue using a capsid mutant AAV8 vector in a mouse model of CNGA3-achromatopsia.PloS one · 2017Article
- The Degeneration and Apoptosis Patterns of Cone Photoreceptors inJournal of ophthalmology · 2017Article
- Effects of Subretinal Gene Transfer at Different Time Points in a Mouse Model of Retinal Degeneration.PloS one · 2016Article
- Promising and delivering gene therapies for vision loss.Vision research · 2015Review
- Trans-Corneal Subretinal Injection in Mice and Its Effect on the Function and Morphology of the Retina.PloS one · 2015Article
- The frequency-response electroretinogram distinguishes cone and abnormal rod function in rd12 mice.PloS one · 2015Article
- Gene therapy using stem cells.Cold Spring Harbor perspectives in medicine · 2014Review
- Stem cells for investigation and treatment of inherited retinal disease.Human molecular genetics · 2014Review
- Retinal gene therapy using adeno-associated viral vectors: multiple applications for a small virus.Human gene therapy · 2014Article
- Selective inhibitors of a PAF biosynthetic enzyme lysophosphatidylcholine acyltransferase 2.Journal of lipid research · 2014Article
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Authors and funding
11 authors at 4 institutions in 2 countries.
Funding
Abstract
purposeThe retinal degeneration 11 (rd11) mouse is a newly discovered, naturally occurring animal model with early photoreceptor dysfunction and rapid rod photoreceptor degeneration followed by cone degeneration. The rd11 mice carry a spontaneous mutation in the lysophosphatidylcholine acyltransferase 1 (Lpcat1) gene. Here, we evaluate whether gene replacement therapy using the fast-acting tyrosine-capsid mutant AAV8 (Y733F) can arrest retinal degeneration and restore retinal function in this model.
methodsThe AAV8 (Y733F)-smCBA-Lpcat1 was delivered subretinally to postnatal day 14 (P14) rd11 mice in one eye only. At 10 weeks after injection, treated rd11 mice were examined by visually-guided behavior, electroretinography (ERG) and spectral domain optical coherence tomography (SD-OCT), and then killed for morphologic and biochemical examination.
resultsSubstantial scotopic and photopic ERG signals were maintained in treated rd11 eyes, whereas untreated eyes in the same animals showed extinguished signals. The SD-OCT (in vivo) and light microscopy (in vitro) showed a substantial preservation of the outer nuclear layer in most parts of the treated retina only. Almost wild-type LPCAT1 expression in photoreceptors with strong rod rhodopsin and M/S cone opsin staining, and normal visually-guided water maze behavioral performances were observed in treated rd11 mice.
conclusionsThe results demonstrate that the tyrosine-capsid mutant AAV8 (Y733F) vector is effective for treating rapidly degenerating models of retinal degeneration and, moreover, is more therapeutically effective than AAV2 (Y444, 500, 730F) vector with the same promoter-cDNA payload. To our knowledge, this is the first demonstration of phenotypic rescue by gene therapy in an animal model of retinal degeneration caused by Lpcat1 mutation.
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