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: | Book |
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Elsevier,
2024-03-01T00:00:00Z.
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| Summary: | 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. |
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| Item Description: | 2667-0097 10.1016/j.gpeds.2024.100134 |