ArticleMolecular therapy : the journal of the American Society of Gene Therapy2020
Intrastromal Gene Therapy Prevents and Reverses Advanced Corneal Clouding in a Canine Model of Mucopolysaccharidosis I.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers.
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Who cites it
23 citing papers in PubMed.
- Topical application of Cas9 ribonucleoproteins inhibits corneal neovascularization in a mouse model of alkali burn injury.Gene therapy · 2026Article
- Clinical response to systemic AAV gene therapy in a large animal model of late-stage lysosomal storage disease.Gene therapy · 2026Article
- Adeno-associated vector corneal gene therapy reverses corneal clouding in a feline model of mucopolysaccharidosis VI.PloS one · 2025Article
- A chimeric anti-vascularization immunomodulator prevents high-risk corneal transplantation rejection via ex vivo gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Sustained and Efficient Delivery of Antivascular Endothelial Growth Factor by the Adeno-associated Virus for the Treatment of Corneal Neovascularization: An Outlook for Its Clinical Translation.Journal of ophthalmology · 2024Review
- rAAV-PHP.B escapes the mouse eye and causes lethality whereas rAAV9 can transduce aniridic corneal limbal stem cells without lethality.Gene therapy · 2023Article
- AAV-mediated expression of HLA-G for the prevention of experimental ocular graft vs. host disease.Molecular therapy. Methods & clinical development · 2023Article
- Review
- Clinical and pathological characterization of ophthalmic disease in a canine model of mucopolysaccharidosis type I.Journal of inherited metabolic disease · 2023Article
- Corneal stromal repair and regeneration.Progress in retinal and eye research · 2022Review
- Ocular Drug Delivery: Advancements and Innovations.Pharmaceutics · 2022Review
- Mucopolysaccharidosis: A broad review.Indian journal of ophthalmology · 2022Review
- Multimodal ocular imaging of known and novel corneal stromal disorders in dogs.BMC veterinary research · 2022Article
- Customized blood-brain barrier shuttle peptide to increase AAV9 vector crossing the BBB and augment transduction in the brain.Biomaterials · 2022Article
- Efficacious, safe, and stable inhibition of corneal neovascularization by AAV-vectored anti-VEGF therapeutics.Molecular therapy. Methods & clinical development · 2021Article
- Management of Corneal Clouding in Patients with Mucopolysaccharidosis.Journal of clinical medicine · 2021Review
- Human MiniPromoters for ocular-rAAV expression in ON bipolar, cone, corneal, endothelial, Müller glial, and PAX6 cells.Gene therapy · 2021Article
- Mucopolysaccharidosis Type I: Current Treatments, Limitations, and Prospects for Improvement.Biomolecules · 2021Review
- Adeno-Associated Virus Vector Mobilization, Risk Versus Reality.Human gene therapy · 2020Article
- Ocular Tolerability and Immune Response to Corneal Intrastromal AAV-Molecular therapy. Methods & clinical development · 2020Article
Corrections and comments
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Authors and funding
11 authors.
Funding
Abstract
Mucopolysaccharidosis type I (MPS I) is an autosomal recessive lysosomal storage disease characterized by severe phenotypes, including corneal clouding. MPS I is caused by mutations in alpha-l-iduronidase (IDUA), a ubiquitous enzyme that catalyzes the hydrolysis of glycosaminoglycans. Currently, no treatment exists to address MPS I corneal clouding other than corneal transplantation, which is complicated by a high risk for rejection. Investigation of an adeno-associated virus (AAV) IDUA gene addition strategy targeting the corneal stroma addresses this deficiency. In MPS I canines with early or advanced corneal disease, a single intrastromal AAV8G9-IDUA injection was well tolerated at all administered doses. The eyes with advanced disease demonstrated resolution of corneal clouding as early as 1 week post-injection, followed by sustained corneal transparency until the experimental endpoint of 25 weeks. AAV8G9-IDUA injection in the MPS I canine eye with early corneal disease prevented the development of advanced corneal changes while restoring clarity. Biodistribution studies demonstrated vector genomes in ocular compartments other than the cornea and in some systemic organs; however, a capsid antibody response was detected in only the highest dosed subject. Collectively, the results suggest that intrastromal AAV8G9-IDUA therapy prevents and reverses visual impairment associated with MPS I corneal clouding.
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