Concurrent progress of reprogramming and gene correction to overcome therapeutic limitation of mutant ALK2-iPSC

Researchers have found a way to reprogram skin cells into stem cells, and fix their genetic flaws at the same time. The standard way of generating induced pluripotent stem cells (iPSCs) that can be used to treat genetic conditions is to reprogram an adult cell, then fix the genetic problem. However, a team led by Sun-ku Chung at the Korea Institute of Oriental Medicine found that didn't work when dealing with fibrodysplasia ossificans progressiva, a condition in which muscle and connective tissue is gradually replaced by bone, because the mutation responsible also disables the iPSCs. Their solution was to combine the reprogramming and gene-editing steps. The researchers suggest this can save time, effort and money not only when dealing with rare diseases but also more generally in gene therapy and disease modeling.