Rescue of ATP7B function in hepatocyte-like cells from Wilson's disease induced pluripotent stem cells using gene therapy or the chaperone drug curcumin. [electronic resource]
- Human molecular genetics Aug 2011
- 3176-87 p. digital
Publication Type: Journal Article; Research Support, Non-U.S. Gov't
1460-2083
10.1093/hmg/ddr223 doi
Adenosine Triphosphatases--genetics Base Sequence Cation Transport Proteins--genetics Copper--metabolism Copper-Transporting ATPases Curcumin--therapeutic use Genetic Therapy Hepatocytes--metabolism Hepatolenticular Degeneration--drug therapy Humans Induced Pluripotent Stem Cells--metabolism Male Middle Aged Molecular Chaperones--therapeutic use Molecular Sequence Data Mutation--genetics Protein Transport Subcellular Fractions--metabolism