Fig. 1: iPSC study designs address different research questions.
From: Power and optimal study design in iPSC-based brain disease modelling
Design 1 (case–control) is characterized by including iPSC lines from multiple independent donors per condition. For design 2A (single isogenic pair), an iPSC line derived from a single donor is gene-edited (or treated), to create two iPSC lines that share the same genetic background and only differ at the gene locus of interest (or treatment). Design 2B is an extension of 2A, in which multiple variants (or treatments) are edited into the same genetic background. In Design 3, multiple isogenic pairs are created. The table represents the ability of each design to answer the research questions described on the left. Green: suitable; Orange: possible, but not optimal; Red: not possible.
