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Therapeutics Development for Alagille Syndrome

Authors :
Wei Zheng
Ivan Pavlinov
Atena Farkhondeh
Steven Rodems
Karsten Baumgaertel
Phillip Sanchez
Source :
Frontiers in Pharmacology, Vol 12 (2021), Frontiers in Pharmacology
Publication Year :
2021
Publisher :
Frontiers Media SA, 2021.

Abstract

Advancements in treatment for the rare genetic disorder known as Alagille Syndrome (ALGS) have been regrettably slow. The large variety of mutations to the JAG1 and NOTCH2 genes which lead to ALGS pose a unique challenge for developing targeted treatments. Due to the central role of the Notch signaling pathway in several cancers, traditional treatment modalities which compensate for the loss in activity caused by mutation are rightly excluded. Unfortunately, current treatment plans for ALGS focus on relieving symptoms of the disorder and do not address the underlying causes of disease. Here we review several of the current and potential key technologies and strategies which may yield a significant leap in developing targeted therapies for this disorder.

Details

Language :
English
ISSN :
16639812
Volume :
12
Database :
OpenAIRE
Journal :
Frontiers in Pharmacology
Accession number :
edsair.doi.dedup.....9c45c97a6df67ea2bea0595975ac6651
Full Text :
https://doi.org/10.3389/fphar.2021.704586