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...

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Detalles Bibliográficos
Autores principales: Chen, Binbin, Altman, Russ B.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2017
Materias:
Research
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

Internet

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

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