Opportunities for developing therapies for rare genetic diseases: focus on gain-of-function and allostery

Opportunities for developing therapies for rare genetic diseases: focus on gain-of-function and allostery

Abstract 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...

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Bibliographic Details
Main Authors: Binbin Chen, Russ B. Altman
Format: Article
Language:English
Published: BMC 2017-04-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Genetic diseases
Rare diseases
Orphan drugs
Drug targets
Gain-of-function
Allosteric
Online Access:http://link.springer.com/article/10.1186/s13023-017-0614-4

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http://link.springer.com/article/10.1186/s13023-017-0614-4

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