Ensuring that drug candidates can reach the clinic is no easy task, so having models that can closely represent human pathology is crucial. Here, Dr Beth Hoffman, CEO of Origami Therapeutics, describes the successes and challenges of using human disease cell models in drug discovery.
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Cystic fibrosis (CF)
A team of scientists used a CRISPR-Cas9 technique known as prime editing to correct cystic fibrosis in cultured human stem cells.