Scientists hsve generated simple kidney-like structures called organoids and used them to identify potential drugs to treat adult-onset polycystic kidney disease. To accelerate the quest for new treatments for ADPKD, researchers used pluripotent stem cells to grow organoids consisting of one or two structures resembling the kidney’s filtering units, known as nephrons. To make the organoids useful for studying ADPKD, the scientists used CRISPR/Cas9 gene editing to inactivate PKD1 or PKD2. As anticipated, the gene-edited organoids began to form cysts. After testing a collection of 247 enzyme inhibitor compounds on the organoids, the scientists found nine that inhibited the growth of the cysts, without stunting the overall growth of the organoids. One compound, quinazoline, was particularly effective.
Scientists use mini-kidney models to identify potential drugs for polycystic kidney disease
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