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Therapeutics Development for Alagille Syndrome

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 signal...

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Autores principales: Sanchez, Phillip, Farkhondeh, Atena, Pavlinov, Ivan, Baumgaertel, Karsten, Rodems, Steven, Zheng, Wei
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8419306/
https://www.ncbi.nlm.nih.gov/pubmed/34497511
http://dx.doi.org/10.3389/fphar.2021.704586
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author Sanchez, Phillip
Farkhondeh, Atena
Pavlinov, Ivan
Baumgaertel, Karsten
Rodems, Steven
Zheng, Wei
author_facet Sanchez, Phillip
Farkhondeh, Atena
Pavlinov, Ivan
Baumgaertel, Karsten
Rodems, Steven
Zheng, Wei
author_sort Sanchez, Phillip
collection PubMed
description 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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spelling pubmed-84193062021-09-07 Therapeutics Development for Alagille Syndrome Sanchez, Phillip Farkhondeh, Atena Pavlinov, Ivan Baumgaertel, Karsten Rodems, Steven Zheng, Wei Front Pharmacol Pharmacology 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. Frontiers Media S.A. 2021-08-23 /pmc/articles/PMC8419306/ /pubmed/34497511 http://dx.doi.org/10.3389/fphar.2021.704586 Text en Copyright © 2021 Sanchez, Farkhondeh, Pavlinov, Baumgaertel, Rodems and Zheng. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Pharmacology
Sanchez, Phillip
Farkhondeh, Atena
Pavlinov, Ivan
Baumgaertel, Karsten
Rodems, Steven
Zheng, Wei
Therapeutics Development for Alagille Syndrome
title Therapeutics Development for Alagille Syndrome
title_full Therapeutics Development for Alagille Syndrome
title_fullStr Therapeutics Development for Alagille Syndrome
title_full_unstemmed Therapeutics Development for Alagille Syndrome
title_short Therapeutics Development for Alagille Syndrome
title_sort therapeutics development for alagille syndrome
topic Pharmacology
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8419306/
https://www.ncbi.nlm.nih.gov/pubmed/34497511
http://dx.doi.org/10.3389/fphar.2021.704586
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