Autosomal dominant polycystic kidney disease (ADPKD), the most common inherited kidney disease, leads to kidney failure in most patients. While mutations in the Polycystin 1 (PKD1) gene cause 85% of cases, we do not fully understand how dysregulation of PKD1 leads to cyst formation on a molecular level.
The four transcription factors that make up the Yamanaka “cocktail” (Oct4, Sox2, Klf4, and Myc, or OSKM) are well known for their capacity to reprogram somatic cells into induced pluripotent stem cells (iPSCs).
Making high‐quality dopamine‐producing cells for basic studies or small molecule screening remains critical to the development of novel therapeutics for ventral midbrain disorders. However, many ventral midbrain assays suffer from low signal to noise ratio thanks to low levels of dopamine and tyrosine hydroxylase, the rate‐limiting enzyme for dopamine synthesis.
While recent studies have demonstrated the generation of human pluripotent stem cell (PSC)-derived midbrain organoids that display structural and functional features of the midbrain, multiple issues currently hinder clinical translation.
The Stem Cells Portal is a shared platform for the STEM CELLS and STEM CELLS Translational Medicine sister journals, providing up-to-the-minute coverage of the latest research from bench science and developments to clinical applications.