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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| Formatua: | Artigo |
| Hizkuntza: | Inglês |
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BMC
2024-08-01
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| Saila: | Journal of Biomedical Science |
| Gaiak: | |
| Sarrera elektronikoa: | https://doi.org/10.1186/s12929-024-01070-1 |
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