Dataset for: Targeting the site encoded by SERPINA1*E342K for treating alpha-1 antitrypsin deficiency associated liver diseases
Posted on 2019-07-31 - 15:43 authored by Wiley Admin
Alpha1-antitrypsin (AAT) deficiency predisposes individuals to emphysema and liver diseases such as cirrhosis and hepatocellular carcinoma. The deficiency results from mutations in the SERPIN1A gene encoding AAT molecules that cause hepatotoxic retention within the endoplasmic reticulum. Since the E342K mutation is the basis for destabilization leading to lung and liver pathology, we used the crystal structure of the mutated AAT as the basis for molecular docking selection of candidate compounds that may bind and stabilize the 342K structural pocket. We identified compounds that inhibited intracellular accumulation of AAT in hepatocytes in vitro. These data suggest that drug binding to a structural site encoded by a mutation associated with AAT deficiency has the potential for clinical utility by modulating conformational transitions.
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Zhang, Xiaojuan; Kien, Pham; Li, Danmeng; Schutte, Ryan; Brantly, Mark; Liu, Chen; et al. (2019). Dataset for: Targeting the site encoded by SERPINA1*E342K for treating alpha-1 antitrypsin deficiency associated liver diseases. Wiley. Collection. https://doi.org/10.6084/m9.figshare.c.4510481.v1
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AUTHORS (8)
XZ
Xiaojuan Zhang
PK
Pham Kien
DL
Danmeng Li
RS
Ryan Schutte
MB
Mark Brantly
CL
Chen Liu
DO
David Ostrov
CATEGORIES
- Animal physiology - biophysics
- Human biophysics
- Synthetic biology
- Biochemistry and cell biology not elsewhere classified
- Plant biology not elsewhere classified
- Virology
- Cell development, proliferation and death
- Bioinformatics and computational biology not elsewhere classified
- Immunology not elsewhere classified
- Neurosciences not elsewhere classified
- Receptors and membrane biology
- Plant cell and molecular biology
- Animal cell and molecular biology
- Evolutionary biology not elsewhere classified
- Signal transduction
- Cancer cell biology
- Systems biology
- Structural biology (incl. macromolecular modelling)
KEYWORDS
alpha-1 antitrypsinliver diseasestructure-based drug discoverymolecular dockingBiophysicsSynthetic BiologyBiochemistryPlant BiologyVirologyCell Development, Proliferation and DeathComputational BiologyImmunologyNeuroscienceReceptors and Membrane BiologyMolecular BiologyEvolutionary BiologySignal TransductionCancer Cell BiologySystems BiologyStructural Biology