New therapeutic modalities for rare diseases
Genetic Diseases affect about 2 % to 3 % of all live births. It has been reported that genetic disorders can account for up to 22 % of infant deaths. Despite this significant effort to create new therapies, treatment is available only for about 5 % of these diseases due to undiscovered etiology or r...
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| Format: | Article |
| Language: | English |
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Elsevier
2024-03-01
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| Series: | Global Pediatrics |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S2667009724000022 |
| _version_ | 1797261801127149568 |
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| author | Fatih S. Ezgu |
| author_facet | Fatih S. Ezgu |
| author_sort | Fatih S. Ezgu |
| collection | DOAJ |
| description | Genetic Diseases affect about 2 % to 3 % of all live births. It has been reported that genetic disorders can account for up to 22 % of infant deaths. Despite this significant effort to create new therapies, treatment is available only for about 5 % of these diseases due to undiscovered etiology or rapid progression to the point of no return. Efforts for new and effective etiology-targeted treatment methods for rare genetic disorders have significantly increased in recent years. Gene-targeted novel treatments such as gene replacement therapy and gene editing, mainly gene-targeted, have significant potential to be the primary treatment modalities in the future. |
| first_indexed | 2024-03-08T15:31:57Z |
| format | Article |
| id | doaj.art-c6f1fc7760034eb583ad2233c2fede08 |
| institution | Directory Open Access Journal |
| issn | 2667-0097 |
| language | English |
| last_indexed | 2024-04-24T23:46:59Z |
| publishDate | 2024-03-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Global Pediatrics |
| spelling | doaj.art-c6f1fc7760034eb583ad2233c2fede082024-03-15T04:44:55ZengElsevierGlobal Pediatrics2667-00972024-03-017100134New therapeutic modalities for rare diseasesFatih S. Ezgu0Departments of Pediatric Metabolic and Genetic Disorders, Gazi University Faculty of Medicine, Ankara, TurkeyGenetic Diseases affect about 2 % to 3 % of all live births. It has been reported that genetic disorders can account for up to 22 % of infant deaths. Despite this significant effort to create new therapies, treatment is available only for about 5 % of these diseases due to undiscovered etiology or rapid progression to the point of no return. Efforts for new and effective etiology-targeted treatment methods for rare genetic disorders have significantly increased in recent years. Gene-targeted novel treatments such as gene replacement therapy and gene editing, mainly gene-targeted, have significant potential to be the primary treatment modalities in the future.http://www.sciencedirect.com/science/article/pii/S2667009724000022GeneticsTreatmentRare disease |
| spellingShingle | Fatih S. Ezgu New therapeutic modalities for rare diseases Global Pediatrics Genetics Treatment Rare disease |
| title | New therapeutic modalities for rare diseases |
| title_full | New therapeutic modalities for rare diseases |
| title_fullStr | New therapeutic modalities for rare diseases |
| title_full_unstemmed | New therapeutic modalities for rare diseases |
| title_short | New therapeutic modalities for rare diseases |
| title_sort | new therapeutic modalities for rare diseases |
| topic | Genetics Treatment Rare disease |
| url | http://www.sciencedirect.com/science/article/pii/S2667009724000022 |
| work_keys_str_mv | AT fatihsezgu newtherapeuticmodalitiesforrarediseases |