In the past decade, human induced pluripotent stem cells (hiPSCs) have emerged as a powerful tool to bring the human biological context earlier into therapeutic development. These cells can self-renew and differentiate into functional cell types which recapitulate human pathophysiology, genetic diversity, and human serotype.
Optimization and efficacy testing of AAVs in a relevant human context before moving into animal models can save time, reduce the number of animals needed and increase success in later stages.
This poster shows how Ncardia developed a hiPSC-derived model for Friedreich's Ataxia (FRDA), a rare, degenerative neuromuscular disorder, and used it as a platform for AAV testing.