Treatment of Inherited Retinal Dystrophies with Somatic Cell Therapy Medicinal Product: A Review
Inherited retinal dystrophies and retinal degenerations related to more common diseases (i.e., age-related macular dystrophy) are a major issue and one of the main causes of low vision in pediatric and elderly age groups. Advancement and understanding in molecular biology and the possibilities raise...
| Main Authors: | , , , , , |
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| Format: | Article |
| Language: | English |
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MDPI AG
2022-05-01
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| Series: | Life |
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| Online Access: | https://www.mdpi.com/2075-1729/12/5/708 |
| _version_ | 1797498502388908032 |
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| author | Giacomo Maria Bacci Valentina Becherucci Elisa Marziali Andrea Sodi Franco Bambi Roberto Caputo |
| author_facet | Giacomo Maria Bacci Valentina Becherucci Elisa Marziali Andrea Sodi Franco Bambi Roberto Caputo |
| author_sort | Giacomo Maria Bacci |
| collection | DOAJ |
| description | Inherited retinal dystrophies and retinal degenerations related to more common diseases (i.e., age-related macular dystrophy) are a major issue and one of the main causes of low vision in pediatric and elderly age groups. Advancement and understanding in molecular biology and the possibilities raised by gene-editing techniques opened a new era for clinicians and patients due to feasible possibilities of treating disabling diseases and the reduction in their complications burden. The scope of this review is to focus on the state-of-the-art in somatic cell therapy medicinal products as the basis of new insights and possibilities to use this approach to treat rare eye diseases. |
| first_indexed | 2024-03-10T03:34:14Z |
| format | Article |
| id | doaj.art-dd61e2a78be24af984b6be22bfd10de0 |
| institution | Directory Open Access Journal |
| issn | 2075-1729 |
| language | English |
| last_indexed | 2024-03-10T03:34:14Z |
| publishDate | 2022-05-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Life |
| spelling | doaj.art-dd61e2a78be24af984b6be22bfd10de02023-11-23T11:50:17ZengMDPI AGLife2075-17292022-05-0112570810.3390/life12050708Treatment of Inherited Retinal Dystrophies with Somatic Cell Therapy Medicinal Product: A ReviewGiacomo Maria Bacci0Valentina Becherucci1Elisa Marziali2Andrea Sodi3Franco Bambi4Roberto Caputo5Pediatric Ophthalmology Unit, Children’s Hospital A. Meyer-University of Florence, 50139 Florence, ItalyCell Factory Meyer, Children’s Hospital A. Meyer-University of Florence, 50139 Florence, ItalyPediatric Ophthalmology Unit, Children’s Hospital A. Meyer-University of Florence, 50139 Florence, ItalyDepartment of Neuroscience, Psychology, Drug Research and Child Health, University of Florence, 50139 Florence, ItalyCell Factory Meyer, Children’s Hospital A. Meyer-University of Florence, 50139 Florence, ItalyPediatric Ophthalmology Unit, Children’s Hospital A. Meyer-University of Florence, 50139 Florence, ItalyInherited retinal dystrophies and retinal degenerations related to more common diseases (i.e., age-related macular dystrophy) are a major issue and one of the main causes of low vision in pediatric and elderly age groups. Advancement and understanding in molecular biology and the possibilities raised by gene-editing techniques opened a new era for clinicians and patients due to feasible possibilities of treating disabling diseases and the reduction in their complications burden. The scope of this review is to focus on the state-of-the-art in somatic cell therapy medicinal products as the basis of new insights and possibilities to use this approach to treat rare eye diseases.https://www.mdpi.com/2075-1729/12/5/708inherited retinal dystrophiesStargardt diseaseretinitis pigmentosaadvanced therapy medicinal productssomatic cell therapy medicinal productsgood manufacturing practices |
| spellingShingle | Giacomo Maria Bacci Valentina Becherucci Elisa Marziali Andrea Sodi Franco Bambi Roberto Caputo Treatment of Inherited Retinal Dystrophies with Somatic Cell Therapy Medicinal Product: A Review Life inherited retinal dystrophies Stargardt disease retinitis pigmentosa advanced therapy medicinal products somatic cell therapy medicinal products good manufacturing practices |
| title | Treatment of Inherited Retinal Dystrophies with Somatic Cell Therapy Medicinal Product: A Review |
| title_full | Treatment of Inherited Retinal Dystrophies with Somatic Cell Therapy Medicinal Product: A Review |
| title_fullStr | Treatment of Inherited Retinal Dystrophies with Somatic Cell Therapy Medicinal Product: A Review |
| title_full_unstemmed | Treatment of Inherited Retinal Dystrophies with Somatic Cell Therapy Medicinal Product: A Review |
| title_short | Treatment of Inherited Retinal Dystrophies with Somatic Cell Therapy Medicinal Product: A Review |
| title_sort | treatment of inherited retinal dystrophies with somatic cell therapy medicinal product a review |
| topic | inherited retinal dystrophies Stargardt disease retinitis pigmentosa advanced therapy medicinal products somatic cell therapy medicinal products good manufacturing practices |
| url | https://www.mdpi.com/2075-1729/12/5/708 |
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