Gene therapy for ultrarare diseases: a geneticist’s perspective
Abstract Gene therapy has made considerable strides in recent years. More than 4000 protein-coding genes have been implicated in more than 6000 genetic diseases; next-generation sequencing has dramatically revolutionized the diagnosis of genetic diseases. Most genetic diseases are considered very ra...
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| Autor principal: | |
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| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
BMC
2024-08-01
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| Col·lecció: | Journal of Biomedical Science |
| Matèries: | |
| Accés en línia: | https://doi.org/10.1186/s12929-024-01070-1 |
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