ArticleMolecular therapy : the journal of the American Society of Gene Therapy2017
Systemic Injection of RPE65-Programmed Bone Marrow-Derived Cells Prevents Progression of Chronic Retinal Degeneration.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
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Who cites it
17 citing papers in PubMed, 22 citations in OpenAlex.
- Age-Related Macular Degeneration: Therapeutic Strategies Based on Stem Cells.Stem cell reviews and reports · 2025Review
- Impact of Donor and Host Age on Systemic Cell Therapy to Treat Age-Related Macular Degeneration.Cells · 2025Article
- β-secretase 1 overexpression by AAV-mediated gene delivery prevents retina degeneration in a mouse model of age-related macular degeneration.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Article
- Maintenance of Enteral ACE2 Prevents Diabetic Retinopathy in Type 1 Diabetes.Circulation research · 2023Article
- Bone marrow-derived mononuclear stem cells in the treatment of retinal degenerations.Neural regeneration research · 2022Review
- Specific mesoderm subset derived from human pluripotent stem cells ameliorates microvascular pathology in type 2 diabetic mice.Science advances · 2022Article
- Characterizing temporal and spatial recruitment of systemically administered RPE65-programmed bone marrow-derived cells to the retina in a mouse model of age-related macular degeneration.Graefe's archive for clinical and experimental ophthalmology = Albrecht von Graefes Archiv fur klinische und experimentelle Ophthalmologie · 2021Article
- Fasting and fasting-mimicking treatment activate SIRT1/LXRα and alleviate diabetes-induced systemic and microvascular dysfunction.Diabetologia · 2021Article
- Cell transplantation to replace retinal ganglion cells faces challenges - the Switchboard Dilemma.Neural regeneration research · 2021Review
- Effects of FTMT Expression by Retinal Pigment Epithelial Cells on Features of Angiogenesis.International journal of molecular sciences · 2020Article
- Mineral-Coated Microparticles Enhance mRNA-Based Transfection of Human Bone Marrow Cells.Molecular therapy. Nucleic acids · 2019Article
- Loss of Angiotensin-Converting Enzyme 2 Exacerbates Diabetic Retinopathy by Promoting Bone Marrow Dysfunction.Stem cells (Dayton, Ohio) · 2018Article
- Enteral Arg-Gln Dipeptide Administration Increases Retinal Docosahexaenoic Acid and Neuroprotectin D1 in a Murine Model of Retinopathy of Prematurity.Investigative ophthalmology & visual science · 2018Article
- Improving the Transduction of Bone Marrow-Derived Cells with an Integrase-Defective Lentiviral Vector.Human gene therapy methods · 2018Article
- Bone Marrow-Derived Cell Recruitment to the Neurosensory Retina and Retinal Pigment Epithelial Cell Layer Following Subthreshold Retinal Phototherapy.Investigative ophthalmology & visual science · 2017Article
- Cell Therapy for Age-Related Macular Degeneration: A New Vision for the Bone Marrow?Molecular therapy : the journal of the American Society of Gene Therapy · 2017Article
- Targeting Lipid Metabolism for the Treatment of Age-Related Macular Degeneration: Insights from Preclinical Mouse Models.Journal of ocular pharmacology and therapeutics : the official journal of the Association for Ocular Pharmacology and TherapeuticsReview
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8 authors at 3 institutions in 1 country.
Funding
Abstract
Bone marrow stem and progenitor cells can differentiate into a range of non-hematopoietic cell types, including retinal pigment epithelium (RPE)-like cells. In this study, we programmed bone marrow-derived cells (BMDCs) ex vivo by inserting a stable RPE65 transgene using a lentiviral vector. We tested the efficacy of systemically administered RPE65-programmed BMDCs to prevent visual loss in the superoxide dismutase 2 knockdown (Sod2 KD) mouse model of age-related macular degeneration. Here, we present evidence that these RPE65-programmed BMDCs are recruited to the subretinal space, where they repopulate the RPE layer, preserve the photoreceptor layer, retain the thickness of the neural retina, reduce lipofuscin granule formation, and suppress microgliosis. Importantly, electroretinography and optokinetic response tests confirmed that visual function was significantly improved. Mice treated with non-modified BMDCs or BMDCs pre-programmed with LacZ did not exhibit significant improvement in visual deficit. RPE65-BMDC administration was most effective in early disease, when visual function and retinal morphology returned to near normal, and less effective in late-stage disease. This experimental paradigm offers a minimally invasive cellular therapy that can be given systemically overcoming the need for invasive ocular surgery and offering the potential to arrest progression in early AMD and other RPE-based diseases.
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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.