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Opportunities for developing therapies for rare genetic diseases: focus on gain-of-function and allostery
BACKGROUND: Advances in next generation sequencing technologies have revolutionized our ability to discover the causes of rare genetic diseases. However, developing treatments for these diseases remains challenging. In fact, when we systematically analyze the US FDA orphan drug list, we find that on...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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BioMed Central
2017
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5392956/ https://www.ncbi.nlm.nih.gov/pubmed/28412959 http://dx.doi.org/10.1186/s13023-017-0614-4 |