The potential of RNA therapeutics, such as small interfering RNA (siRNA), messenger RNA (mRNA), antisense oligonucleotides (ASOs), and microRNA (miRNA), can be hindered by low specificity, risk of off-target effects, challenging delivery or limited clinical translation.
Human induced pluripotent stem cell (iPSC) technology offers a powerful platform for addressing those key challenges by bringing in translational disease models and screening assays. Additionally, iPSC-derived cells can be patient-specific, a key aspect to success in the development of therapies for rare diseases.
