The discovery in 2007 that cells isolated from mature human tissues can be reprogrammed into cells that show pluripotency was a major breakthrough in stem cell science.
These hiPSCs under the right culture conditions will differentiate into a wide range of cell types. This technology enables the supply of unlimited quantities of tissues from healthy individuals or patients creating in-vitro models that are more representative of the in-vivo human physiology and disease mechanisms than current models.
Since 2007 research using hiPSC derived cells has accelerated as groups have explored their application in drug discovery and regenerative medicine. Using non-integrating virus vectors such as Sendai, essentially genetically identical cells can be produced from healthy donors or patients. For patients with diseases caused by known genetic mutations it has been shown that hiPSCs differentiated into somatic cells exhibit the disease phenotype. This has even been achieved for some idiopathic diseases such as diabetes and some forms of Alzheimer’s.
This approach has now been used for a wide range of diseases including Parkinson’s, a range of cardiomyopathies, retinitis pigmentosa and Gaucher’s. This technique enables disease models to be used to investigate the mechanisms of disease and to screen compounds for new lead compounds.
The team behind Newcells Biotech has successfully demonstrated the application of Sendai virus produced iPSC lines to recreate the phenotypes of many diseases such as hypoplastic left heart syndrome, XLF deficiency and chronic granulomatous disease.