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Induced Pluripotent Stem Cell (iPSC)

Biopharmaceutical Glossary

Induced Pluripotent Stem Cell (iPSC) designates adult somatic cells reprogrammed to embryonic stem cell-like pluripotent states capable of differentiating into virtually any cell type, achieved through introducing defined transcription factors including Oct4, Sox2, Klf4, and c-Myc. This breakthrough technology recognised with the 2012 Nobel Prize revolutionised regenerative medicine and disease modelling.

The biopharmaceutical industry leverages iPSC technology across diverse applications. Disease modelling employs patient-derived iPSCs differentiating into affected cell types recreating disease features enabling mechanistic studies and drug screening. Drug discovery uses iPSC-derived cells including cardiomyocytes for cardiotoxicity assessment, hepatocytes for metabolism studies, and neurons for neurodegenerative disease research. Cell therapy development differentiates iPSCs into specific cell types for transplantation, with clinical trials underway for retinal disorders, heart disease, and neurological conditions. Safety concerns address residual undifferentiated cells potentially forming teratomas. As reprogramming methods improve and differentiation protocols become more efficient, iPSC applications expand through clinical translation and personalised medicine approaches.

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