期刊
FRONTIERS IN PHARMACOLOGY
卷 12, 期 -, 页码 -出版社
FRONTIERS MEDIA SA
DOI: 10.3389/fphar.2021.704586
关键词
alagille syndrome; JAG1; notch signaling pathway; liver; bile duct; drug development
资金
- Intramural Research Programs of the National Center for Advancing Translational Sciences (NCATS), National Institutes of Health [ZIA TR000018-06]
Advancements in treating the rare genetic disorder known as Alagille Syndrome (ALGS) have been slow due to the variety of mutations in the JAG1 and NOTCH2 genes. Current treatment plans focus on relieving symptoms rather than addressing the underlying causes of the disease. Developing targeted therapies for ALGS is challenging due to the central role of the Notch signaling pathway in cancer.
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.
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