Generation of iPSC lines with SLC16A2:G401R or SLC16A2 knock out

Summary: CRISPR-Cas9 technology combined with human induced pluripotent stem cells enables precise disease modeling in pluripotent and specialized cells. This article presents an optimized CRISPR-Cas9 pipeline called ASSURED (affordable, successful, specific, user-friendly, rapid, efficient, and deliverable) for generating gene-modified single-cell-derived knockout or single-nucleotide-polymorphism-modified knockin hiPSC clones. The article describes the steps for analyzing targeted genomic sequences, designing guide RNAs and homology repair templates, as well as the CRISPR-Cas9 delivery workflow, efficiency evaluation, automated cell isolation, and clone screening.

Summary: Triac shows sustainable relief of key features in MCT8 deficiency patients of all ages, highlighting its real-life potential for MCT8 deficiency.