Rare Genetic Disorders: Novel Treatment Strategies and Insights Into Human Biology

The last decade has seen a dramatic increase in innovative ideas for the treatment of genetic disorders for which no curative therapies exist. Gene and protein replacement therapies stand out as novel approaches to treat a select group of these diseases, such as certain tissue fragility disorders. F...

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Bibliographic Details
Main Authors: Peter J. Koch, Maranke I. Koster
Format: Article
Language:English
Published: Frontiers Media S.A. 2021-08-01
Series:Frontiers in Genetics
Subjects:
Online Access:https://www.frontiersin.org/articles/10.3389/fgene.2021.714764/full