Therapeutics Development for Alagille Syndrome

• Principais autores: Phillip Sanchez (Autor), Atena Farkhondeh (Autor), Ivan Pavlinov (Autor), Karsten Baumgaertel (Autor), Steven Rodems (Autor), Wei Zheng (Autor)
• Formato: Livro
• Publicado em: Frontiers Media S.A., 2021-08-01T00:00:00Z.
• Assuntos: article
• Acesso em linha: Connect to this object online.

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...