Advanced Therapy Medicinal Products for Eye Diseases: Goals and Challenges
The concept of advanced therapy medicinal products (ATMPs) encompasses novel kinds of medicines for human use that are based on genes, cells or tissues. These intend to offer not only regeneration, but complete functional recovery of diseased tissues and organs using different strategies. Gene thera...
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| Aineistotyyppi: | Online |
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| Kieli: | englanti |
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MDPI - Multidisciplinary Digital Publishing Institute
2022
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| Linkit: | ONIX_20220111_9783036526119_959 |
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| description | The concept of advanced therapy medicinal products (ATMPs) encompasses novel kinds of medicines for human use that are based on genes, cells or tissues. These intend to offer not only regeneration, but complete functional recovery of diseased tissues and organs using different strategies. Gene therapy, cell therapy and tissue engineering are the main areas in which promising advanced therapies are emerging. The eye is a very complex organ whose main structures, the cornea and the retina, play a pivotal role in maintaining normal vision, as severe alterations in these tissues can lead to blindness. Ocular tissues are starting to benefit from ATMPs by fighting against the enormous complexity and devastating potential of many ocular diseases. However, developments arising from this field of work face important challenges related to vectors to deliver drugs and genetic material to target tissues, suitable biomaterials to prepare cell scaffolds and cell stemness, among others—not to mention the complicated legislation around ATMPs, the complexity in production and quality control and the absence of standardized protocols.The purpose of this Special Issue is to serve as an overview of the current progress in the application of cell and gene therapies, as well as tissue engineering to restore functionality in diseased ocular structures, and the challenges linked to reaching patients. |
| format | Online |
| id | doab-20.500.12854ir-77128 |
| institution | Directory of Open Access Books |
| language | eng |
| publishDate | 2022 |
| publishDateRange | 2022 |
| publishDateSort | 2022 |
| publisher | MDPI - Multidisciplinary Digital Publishing Institute |
| publisherStr | MDPI - Multidisciplinary Digital Publishing Institute |
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| spelling | doab-20.500.12854ir-771282024-03-31T13:09:01Z Advanced Therapy Medicinal Products for Eye Diseases: Goals and Challenges Diebold, Yolanda García-Posadas, Laura MPC polymer dry eye ocular surface lacrimal fluid mucin 3D bioprinting cornea retina ophthalmology tissue regeneration electrospinning conjunctiva decellularized tissue matrix small intestinal submucosa urinary bladder matrix polycaprolactone fiber tissue engineering stratification conjunctival epithelial cells hydrogel keratoplasty scaffold collagen advanced therapy medicinal product ATMP cell therapy gene therapy eye ocular regulatory marketing authorization double-crosslinking carbodiimide glutaraldehyde sodium metabisulfite sodium borohydride EDC/NHS stem cells retinal diseases optic nerve diseases cell replacement cell sources advanced therapies ocular mucosa blindness CLET limbal niche limbal stem cell LSCD mesenchymal stem cell transplantation MSCT corneal limbus decellularized xenograft recellularization mesenchymal stem cells n/a thema EDItEUR::M Medicine and Nursing The concept of advanced therapy medicinal products (ATMPs) encompasses novel kinds of medicines for human use that are based on genes, cells or tissues. These intend to offer not only regeneration, but complete functional recovery of diseased tissues and organs using different strategies. Gene therapy, cell therapy and tissue engineering are the main areas in which promising advanced therapies are emerging. The eye is a very complex organ whose main structures, the cornea and the retina, play a pivotal role in maintaining normal vision, as severe alterations in these tissues can lead to blindness. Ocular tissues are starting to benefit from ATMPs by fighting against the enormous complexity and devastating potential of many ocular diseases. However, developments arising from this field of work face important challenges related to vectors to deliver drugs and genetic material to target tissues, suitable biomaterials to prepare cell scaffolds and cell stemness, among others—not to mention the complicated legislation around ATMPs, the complexity in production and quality control and the absence of standardized protocols.The purpose of this Special Issue is to serve as an overview of the current progress in the application of cell and gene therapies, as well as tissue engineering to restore functionality in diseased ocular structures, and the challenges linked to reaching patients. 2022-01-11T13:53:06Z 2022-01-11T13:53:06Z 2021 book ONIX_20220111_9783036526119_959 9783036526119 9783036526102 https://directory.doabooks.org/handle/20.500.12854/77128 eng image/jpeg Attribution 4.0 International https://mdpi.com/books/pdfview/book/4746 https://mdpi.com/books/pdfview/book/4746 MDPI - Multidisciplinary Digital Publishing Institute 10.3390/books978-3-0365-2610-2 10.3390/books978-3-0365-2610-2 46cabcaa-dd94-4bfe-87b4-55023c1b36d0 7224c55a-d5e7-455a-a8c7-fed603648816 9783036526119 9783036526102 207 Basel, Switzerland [grantnumber unknown] open access |
| spellingShingle | MPC polymer dry eye ocular surface lacrimal fluid mucin 3D bioprinting cornea retina ophthalmology tissue regeneration electrospinning conjunctiva decellularized tissue matrix small intestinal submucosa urinary bladder matrix polycaprolactone fiber tissue engineering stratification conjunctival epithelial cells hydrogel keratoplasty scaffold collagen advanced therapy medicinal product ATMP cell therapy gene therapy eye ocular regulatory marketing authorization double-crosslinking carbodiimide glutaraldehyde sodium metabisulfite sodium borohydride EDC/NHS stem cells retinal diseases optic nerve diseases cell replacement cell sources advanced therapies ocular mucosa blindness CLET limbal niche limbal stem cell LSCD mesenchymal stem cell transplantation MSCT corneal limbus decellularized xenograft recellularization mesenchymal stem cells n/a thema EDItEUR::M Medicine and Nursing Advanced Therapy Medicinal Products for Eye Diseases: Goals and Challenges |
| title | Advanced Therapy Medicinal Products for Eye Diseases: Goals and Challenges |
| title_full | Advanced Therapy Medicinal Products for Eye Diseases: Goals and Challenges |
| title_fullStr | Advanced Therapy Medicinal Products for Eye Diseases: Goals and Challenges |
| title_full_unstemmed | Advanced Therapy Medicinal Products for Eye Diseases: Goals and Challenges |
| title_short | Advanced Therapy Medicinal Products for Eye Diseases: Goals and Challenges |
| title_sort | advanced therapy medicinal products for eye diseases goals and challenges |
| topic | MPC polymer dry eye ocular surface lacrimal fluid mucin 3D bioprinting cornea retina ophthalmology tissue regeneration electrospinning conjunctiva decellularized tissue matrix small intestinal submucosa urinary bladder matrix polycaprolactone fiber tissue engineering stratification conjunctival epithelial cells hydrogel keratoplasty scaffold collagen advanced therapy medicinal product ATMP cell therapy gene therapy eye ocular regulatory marketing authorization double-crosslinking carbodiimide glutaraldehyde sodium metabisulfite sodium borohydride EDC/NHS stem cells retinal diseases optic nerve diseases cell replacement cell sources advanced therapies ocular mucosa blindness CLET limbal niche limbal stem cell LSCD mesenchymal stem cell transplantation MSCT corneal limbus decellularized xenograft recellularization mesenchymal stem cells n/a thema EDItEUR::M Medicine and Nursing |
| topic_facet | MPC polymer dry eye ocular surface lacrimal fluid mucin 3D bioprinting cornea retina ophthalmology tissue regeneration electrospinning conjunctiva decellularized tissue matrix small intestinal submucosa urinary bladder matrix polycaprolactone fiber tissue engineering stratification conjunctival epithelial cells hydrogel keratoplasty scaffold collagen advanced therapy medicinal product ATMP cell therapy gene therapy eye ocular regulatory marketing authorization double-crosslinking carbodiimide glutaraldehyde sodium metabisulfite sodium borohydride EDC/NHS stem cells retinal diseases optic nerve diseases cell replacement cell sources advanced therapies ocular mucosa blindness CLET limbal niche limbal stem cell LSCD mesenchymal stem cell transplantation MSCT corneal limbus decellularized xenograft recellularization mesenchymal stem cells n/a thema EDItEUR::M Medicine and Nursing |
| url | ONIX_20220111_9783036526119_959 |