Stem Cells Checks Novel Therapies

Efficiency of drug therapies against genetic disorders, like Gaucher disease can be ensured by using stem cells as model. Researchers at university of Maryland school of Medicine has reconditioned stem cells to develop into cells which are geneticallysimilar and react against drugs in a similar way as cells from patients with Gaucher disease. With this scientist can check the potential of novel therapies and give a boost up to the field of drug discovery.
Gaucher disease is a common lipid storage disease, commonly found in Ashkenazi Jews. Disease have three types, type 1, 2 and 3 ranging from mildest to fatal. It’s a genetic disorder occurring in recessive condition with both parents as carrier.
Scientist engineered skin cells from gaucher patient to produce human induced pluripotent stem cells which were further differentiated into macrophages and neuronal cells. In Gaucher disease, macrophages are inefficient to digest RBC due to lack of lipid digestion present in RBC membrane  which leads to the blockage of membrane transport pathways in the macrophages settled in the bone marrow, spleen and liver. Similar macrophages were developed by the scientists from the engineered stem cells which were reconfirmed by a recombinant enzyme, effective in treating type 1 gaucher disease. Macrophages resumed their function on treatment with the desired enzyme.
This research in field of stem cells will speed up the avenues in field of drug discovery and pave new pathways for genetic disease treatment.