Cell-Based Small-Molecule Screening Identifying Proteostasis Regulators Enhancing Factor VIII Missense Mutant Secretion

Cell-Based Small-Molecule Screening Identifying Proteostasis Regulators Enhancing Factor VIII Missense Mutant Secretion

Missense mutations are the most prevalent alterations in genetic disorders such as hemophilia A (HA), which results from coagulation factor VIII (FVIII) deficiencies. These mutations disrupt protein biosynthesis, folding, secretion, and function. Current treatments for HA are extremely expensive and...

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Bibliographic Details
Main Authors: Vishal Srivastava, Zhigang Liu, Wei Wei, Yuan Zhang, James C. Paton, Adrienne W. Paton, Tingwei Mu, Bin Zhang
Format: Article
Language:English
Published: MDPI AG 2025-03-01
Series:Biomolecules
Subjects:
factor VIII
secretion
Vorinostat
proteostasis regulators
BiP/GRP78
Online Access:https://www.mdpi.com/2218-273X/15/4/458
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