Ocular Gene Therapy with Adeno-associated Virus Vectors: Current Outlook for Patients and Researchers
In this “Perspective”, we discuss ocular gene therapy – the patient's perspective, the various strategies of gene replacement and gene editing, the place of adeno-associated virus vectors, routes of delivery to the eye and the remaining question - “why does immunity continue to limit efficacy?”...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
| Published: |
Knowledge E
2020-08-01
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| Series: | Journal of Ophthalmic & Vision Research |
| Online Access: | https://doi.org/10.18502/jovr.v15i3.7457 |
| _version_ | 1811255220591656960 |
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| author | Geoffrey A. Casey Kimberly M. Papp Ian M. MacDonald |
| author_facet | Geoffrey A. Casey Kimberly M. Papp Ian M. MacDonald |
| author_sort | Geoffrey A. Casey |
| collection | DOAJ |
| description | In this “Perspective”, we discuss ocular gene therapy – the patient's perspective, the various strategies of gene replacement and gene editing, the place of adeno-associated virus vectors, routes of delivery to the eye and the remaining question - “why does immunity continue to limit efficacy?” Through the coordinated efforts of patients, researchers, granting agencies and industry, and after many years of pre-clinical studies, biochemical, cellular, and animal models, we are seeing clinical trials emerge for many previously untreatable heritable ocular disorders. The pathway to therapies has been led by the successful treatment of the RPE65 form of Leber congenital amaurosis with LUXTURNA
TM
. In some cases, immune reactions to the vectors continue to occur, limiting efficacy. The underlying mechanisms of inflammation require further study, and new vectors need to be designed that limit the triggers of immunity. Researchers studying ocular gene therapies and clinicians enrolling patients in clinical trials must recognize the current limitations of these therapies to properly manage expectations and avoid disappointment, but we believe that gene therapies are well on their way to successful, widespread utilization to treat heritable ocular disorders. |
| first_indexed | 2024-04-12T17:21:40Z |
| format | Article |
| id | doaj.art-6208c28e228c43d7904d4337b0f735e7 |
| institution | Directory Open Access Journal |
| issn | 2008-322X |
| language | English |
| last_indexed | 2024-04-12T17:21:40Z |
| publishDate | 2020-08-01 |
| publisher | Knowledge E |
| record_format | Article |
| series | Journal of Ophthalmic & Vision Research |
| spelling | doaj.art-6208c28e228c43d7904d4337b0f735e72022-12-22T03:23:28ZengKnowledge EJournal of Ophthalmic & Vision Research2008-322X2020-08-0115339639910.18502/jovr.v15i3.7457jovr.v15i3.7457Ocular Gene Therapy with Adeno-associated Virus Vectors: Current Outlook for Patients and ResearchersGeoffrey A. Casey0Kimberly M. Papp1Ian M. MacDonald2 Department of Ophthalmology, Faculty of Medicine and Dentistry, University of Alberta, Canada Department of Ophthalmology, Faculty of Medicine and Dentistry, University of Alberta, Canada Department of Ophthalmology, Faculty of Medicine and Dentistry, University of Alberta, CanadaIn this “Perspective”, we discuss ocular gene therapy – the patient's perspective, the various strategies of gene replacement and gene editing, the place of adeno-associated virus vectors, routes of delivery to the eye and the remaining question - “why does immunity continue to limit efficacy?” Through the coordinated efforts of patients, researchers, granting agencies and industry, and after many years of pre-clinical studies, biochemical, cellular, and animal models, we are seeing clinical trials emerge for many previously untreatable heritable ocular disorders. The pathway to therapies has been led by the successful treatment of the RPE65 form of Leber congenital amaurosis with LUXTURNA TM . In some cases, immune reactions to the vectors continue to occur, limiting efficacy. The underlying mechanisms of inflammation require further study, and new vectors need to be designed that limit the triggers of immunity. Researchers studying ocular gene therapies and clinicians enrolling patients in clinical trials must recognize the current limitations of these therapies to properly manage expectations and avoid disappointment, but we believe that gene therapies are well on their way to successful, widespread utilization to treat heritable ocular disorders.https://doi.org/10.18502/jovr.v15i3.7457 |
| spellingShingle | Geoffrey A. Casey Kimberly M. Papp Ian M. MacDonald Ocular Gene Therapy with Adeno-associated Virus Vectors: Current Outlook for Patients and Researchers Journal of Ophthalmic & Vision Research |
| title | Ocular Gene Therapy with Adeno-associated Virus Vectors: Current Outlook for Patients and Researchers |
| title_full | Ocular Gene Therapy with Adeno-associated Virus Vectors: Current Outlook for Patients and Researchers |
| title_fullStr | Ocular Gene Therapy with Adeno-associated Virus Vectors: Current Outlook for Patients and Researchers |
| title_full_unstemmed | Ocular Gene Therapy with Adeno-associated Virus Vectors: Current Outlook for Patients and Researchers |
| title_short | Ocular Gene Therapy with Adeno-associated Virus Vectors: Current Outlook for Patients and Researchers |
| title_sort | ocular gene therapy with adeno associated virus vectors current outlook for patients and researchers |
| url | https://doi.org/10.18502/jovr.v15i3.7457 |
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