Neural stem cells for disease modeling and evaluation of therapeutics for Tay-Sachs disease.

Mylinh VuRong LiAmanda BaskfieldBilly LuAtena FarkhondehKirill GorshkovOmid MotabarJeanette BeersGuokai ChenJizhong ZouAngela J Espejo-MojicaAlexander Rodríguez-LópezCarlos J Alméciga-DíazLuis A BarreraXuntian JiangDaniel S OryJuan J MaruganWei Zheng

Published in: Orphanet journal of rare diseases (2018)

Our results demonstrate that the Tay-Sachs disease NSCs possess the characteristic phenotype to serve as a cell-based disease model for study of the disease pathogenesis and evaluation of drug efficacy. The enzyme replacement therapy with recombinant Hex A protein and two small molecules (cyclodextrin and tocopherol) significantly ameliorated lipid accumulation in the Tay-Sachs disease cell model.

Keyphrases

cell therapy
mesenchymal stem cells
bone marrow
smoking cessation
protein protein
binding protein
cell free