Krishanu Saha, PhD – Slide of the Week

We present and characterize a robust method for rapid, scarless introduction or correction of disease-associated variants in hPSCs using CRISPR/Cas9. Utilizing non-integrated plasmid vectors that express a puromycin N-acetyl-transferase (PAC) gene, whose expression and translation is linked to that of Cas9, we transiently select for cells based on their early levels of Cas9 protein.

UW-Madison biomanufacturer offers essential gene-transfer capacity

Adeno-associated virus (AAV) is used to insert replacement genes, and its role in genetic therapy trials is expanding quickly, says Carl Ross, managing director of Waisman Biomanufacturing. The AAV production reflects the Waisman lab’s growing importance in the biopharma business, as it’s the only facility on campus meeting FDA “good manufacturing practices” rules for large-scale manufacturing of biological therapies.