Professor Juan Carlos Izpisua Belmonte, co–first author Jun Wu and collaborators have corrected a disease-causing mutation in early stage human embryos. The technique, which uses the CRISPR-Cas9 system, corrected the mutation for a common heart condition called hypertrophic cardiomyopathy at the earliest stage of embryonic development so that the defect would not be passed on to future generations. The scientists were surprised by just how safe and efficient the method was. Not only did a high percentage of embryonic cells get repaired, but also gene correction didn’t induce any detectable off-target mutations and genome instability— major concerns for gene editing. In addition, the researchers developed a robust strategy to ensure the repair occurred consistently in all the cells of the embryo.
- Harnessing plants for the futureGet ready, sunny San Diego: Winter is coming. We’re not talking about a new season of Game of Thrones—although the story is one of epic proportions and high stakes.
- Sung Han was destined for SalkHan, an assistant professor in the Clayton Foundation Laboratories for Peptide Biology, arrived at the Institute a year ago to study how the brain recognizes aversive sensory signals.
- All roads lead to science for Elena Blanco-SuárezFrom early on, it was pretty much a given that Elena Blanco-Suárez would be a scientist.