ArticleMolecular therapy : the journal of the American Society of Gene Therapy2020
Repair of Retinal Degeneration following Ex Vivo Minicircle DNA Gene Therapy and Transplantation of Corrected Photoreceptor Progenitors.
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 19 papers.
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Who cites it
19 citing papers in PubMed, 24 citations in OpenAlex.
- Progress and prospect of minicircle as a minimized non-viral DNA vector in gene therapy and regenerative medicine.Molecular therapy. Nucleic acids · 2025Review
- Animal models for the evaluation of retinal stem cell therapies.Progress in retinal and eye research · 2025Review
- Review
- Cellular component transfer between photoreceptor cells of the retina.Progress in retinal and eye research · 2025Review
- Bioengineering strategies for restoring vision.Nature biomedical engineering · 2023Review
- Progress of iPS cell-based transplantation therapy for retinal diseases.Japanese journal of ophthalmology · 2023Review
- Re-formation of synaptic connectivity in dissociated human stem cell-derived retinal organoid cultures.Proceedings of the National Academy of Sciences of the United States of America · 2023Article
- Minicircle Delivery to the Neural Retina as a Gene Therapy Approach.International journal of molecular sciences · 2022Review
- Subretinal Injection Techniques for Retinal Disease: A Review.Journal of clinical medicine · 2022Review
- CRISPR DNA Base Editing Strategies for Treating Retinitis Pigmentosa Caused by Mutations inGenes · 2022Article
- Retinitis Pigmentosa: Progress in Molecular Pathology and Biotherapeutical Strategies.International journal of molecular sciences · 2022Review
- Outer Retinal Cell Replacement: Putting the Pieces Together.Translational vision science & technology · 2021Review
- Pluripotent stem cell therapy for retinal diseases.Annals of translational medicine · 2021Review
- Genome-Editing Strategies for Treating Human Retinal Degenerations.Human gene therapy · 2021Review
- METTL3-Mediated mStem cells international · 2021Article
- Coculture techniques for modeling retinal development and disease, and enabling regenerative medicine.Stem cells translational medicine · 2020Review
- Improving therapeutic potential of non-viral minimized DNA vectors.Cell & gene therapy insights · 2020Article
- Personalized stem cell-based therapy for degenerative retinal diseases.Stem cell reviews and reports · 2020Article
- Novel Therapeutic Approaches for the Treatment of Retinal Degenerative Diseases: Focus on CRISPR/Cas-Based Gene Editing.Frontiers in neuroscience · 2020Review
Corrections and comments
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Authors and funding
7 authors at 3 institutions in 3 countries.
Funding
Abstract
The authors describe retinal reconstruction and restoration of visual function in heritably blind mice missing the rhodopsin gene using a novel method of ex vivo gene therapy and cell transplantation. Photoreceptor precursors with the same chromosomal genetic mutation were treated ex vivo using minicircle DNA, a non-viral technique that does not present the packaging limitations of adeno-associated virus (AAV) vectors. Following transplantation, genetically modified cells reconstructed a functional retina and supported vision in blind mice harboring the same founder gene mutation. Gene delivery by minicircles showed comparable long-term efficiency to AAV in delivering the missing gene, representing the first non-viral system for robust treatment of photoreceptors. This important proof-of-concept finding provides an innovative convergence of cell and gene therapies for the treatment of hereditary neurodegenerative disease and may be applied in future studies toward ex vivo correction of patient-specific cells to provide an autologous source of tissue to replace lost photoreceptors in inherited retinal blindness. This is the first report using minicircles in photoreceptor progenitors and the first to transplant corrected photoreceptor precursors to restore vision in blind animals.
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Registered trials
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.