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Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina
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Adeno-associated viral vectors reconstitute large proteins in the retina via intein-mediated protein trans-splicing.
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“Upon subretinal AAV administration, split inteins catalyze the seamless joining of the two halves of the ABCA4 protein, thus reconstituting a functional full-length ABCA4 protein in photoreceptors, at levels that largely exceed those achieved via AAV genome recombination by alternative dual AAV-based strategies (5), resulting in therapeutic efficacy in Abca4−/− mice (5).”
From Retinal gene therapy for Stargardt disease with dual AAV intein vectors is both safe and effective in large animal models (2025) · cites this paper as [5]
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“resulting in therapeutic efficacy in Abca4−/− mice (5).”
From Retinal gene therapy for Stargardt disease with dual AAV intein vectors is both safe and effective in large animal models (2025) · cites this paper as [5]
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“at levels that largely exceed those achieved via AAV genome recombination by alternative dual AAV-based strategies (5), resulting in therapeutic efficacy in Abca4−/− mice (5).”
From Retinal gene therapy for Stargardt disease with dual AAV intein vectors is both safe and effective in large animal models (2025) · cites this paper as [5]
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“To overcome this limitation, we have recently developed a dual AAV gene therapy approach, which we named “AAV-ABCA4-intein” vectors (5).”
From Retinal gene therapy for Stargardt disease with dual AAV intein vectors is both safe and effective in large animal models (2025) · cites this paper as [5]
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“Overall, these data show a good safety and expression profile of AAV8.ABCA4.intein vectors in the NHP retina, confirming the promising data from a previous study in mouse models (5).”
From Retinal gene therapy for Stargardt disease with dual AAV intein vectors is both safe and effective in large animal models (2025) · cites this paper as [5]
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“Although some of these studies have revealed amelioration of retinal phenotypes in STGD1 mouse models [29,64,102], the low transduction efficiencies as well as the aberrant production of truncated proteins from single AAV vectors have been two major limitations [103,104].”
From Circumventing the packaging limit of AAV-mediated gene replacement therapy for neurological disorders (2022) · cites this paper as [64]
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“Recently, the Auricchio lab explored the potentials of protein trans-splicing for inherited retinal disorders, proving this technology to achieve superior reassembly efficiencies as compared to other dual AAV vector strategies [64].”
From Circumventing the packaging limit of AAV-mediated gene replacement therapy for neurological disorders (2022) · cites this paper as [64]
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“Finally, the use of inteins has also been explored at the experimental level: AAV2/8-CEP290 protein trans-splicing vectors have achieved reconstitution of the large CEP290 protein in the mouse retina, showing ameliorated retinal phenotypes [64].”
From Circumventing the packaging limit of AAV-mediated gene replacement therapy for neurological disorders (2022) · cites this paper as [64]
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Self-citationKaltak, M., Blanco-Garavito, R., Molday, L. L., Dhaenens, C., Souied, E. H., Platenburg, G. J., Swildens, J., Molday, R. S., & Cremers, F. P. M. (2023) · Figshare · doi:10.6084/m9.figshare.23972916
Self-citationKaltak, M., Blanco-Garavito, R., Molday, L. L., Dhaenens, C., Souied, E. H., Platenburg, G. J., Swildens, J., Molday, R. S., & Cremers, F. P. M. (2023) · Figshare · doi:10.6084/m9.figshare.23972919
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Metadata
- Authors
- Tornabene, P., Trapani, I., Minopoli, R., Centrulo, M., Lupo, M., de Simone, S., Tiberi, P., Dell’Aquila, F., Marrocco, E., Iodice, C., Iuliano, A., Gesualdo, C., Rossi, S., Giaquinto, L., Albert, S., Hoyng, C. B., Polishchuk, E., Cremers, F. P. M., Surace, E. M., Simonelli, F., De Matteis, M. A., Polishchuk, R., & Auricchio, A.
- Affiliations
- Telethon Institute Of Genetics And Medicine (IT)
- Federico II University Hospital (IT)
- Telethon Institute of Genetics and Medicine (TIGEM), 80078 Pozzuoli, Italy.
- University of Campania "Luigi Vanvitelli" (IT)
- Department of Human Genetics and Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, 6525 Nijmegen, Netherlands.
- Radboud University Nijmegen (NL)
- Corresponding author
- Tornabene, Patrizia
- Trapani, Ivana
- Auricchio, Alberto
- ORCID
- Tornabene, Patrizia
- Trapani, Ivana
- Minopoli, Renato
- Lupo, Mariangela
- Tiberi, Paola
- Dell’Aquila, Fabio
- Marrocco, Elena
- Iuliano, Antonella
- Gesualdo, Carlo
- Rossi, Settimio
- Giaquinto, Laura
- Hoyng, Carel B.
- Polishchuk, Elena
- Cremers, Frans P. M.
- Surace, Enrico M.
- Simonelli, Francesca
- De Matteis, Maria A.
- Polishchuk, Roman
- Auricchio, Alberto
- Venue
- Science Translational Medicine
- Published
- 2019-05-15
- Volume
- 11
- Issue
- 492
- ISSN
- 1946-6234 (ISSN-L)
- 1946-6242
- PMC
- PMC6863751
- OpenAlex
- W2945064781
- Cited by
- 192
- References
- 53
- Citations by year
- 2026: 23 · 2025: 40 · 2024: 26 · 2023: 27 · 2022: 28 · 2021: 23 · 2020: 20 · 2019: 5
- FWCI
- 11.41
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Science Translational Medicine (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- CINECA IRIS Institutional Research Information System (University of Basilicata) (submittedVersion, public-domain)
- Archivio Istituzionale della Ricerca - Università degli Studi della Campania "Luigi Vanvitelli" (submittedVersion)
- Radboud Repository (Radboud University) (submittedVersion)
- Zenodo (CERN European Organization for Nuclear Research) (submittedVersion, cc-by)
- Funders
- Universita degli Studi di Napoli Federico II (STAR Program)
- European Research Council (694323)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Health Sciences › Medicine › Ophthalmology › Retinal Development and Disorders
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- split intein, adeno-associated virus vectors, retinal gene therapy, subretinal delivery, inherited retinal disease, inherited blindness, retinal organoids, porcine model
- MeSH terms
- Dependovirus, Animals, Genetic Vectors, Humans, Organoids, Phenotype, Retina, Swine, Gene Transfer Techniques, Trans-Splicing, Photoreceptor Cells, Vertebrate, Inteins, Green Fluorescent Proteins, Mice, Induced Pluripotent Stem Cells
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