October 19, 2020

The Niche

Knoepfler lab stem cell blog

gene editing mitochondrial disease

4 min read

The FDA sent a stern letter today to Dr. John Zhang, PhD, MD, the physician who last year created a genetically modified human embryo in the U.S. and then exported it to Mexico where he self-reported creating a genetically modified baby boy. While the genetically modified baby created in Mexico was reportedly seemingly OK, it’s not clear what kind of health issues he might face in the future and I haven’t seen much data on him. I believe his parents subsequently declined to have him participate …Read More

5 min read

The Juan Carlos Izpisua Belmonte group published a Cell paper today on using gene editing to reverse mutations associated with human mitochondrial disease. The paper is Reddy, et al. and is entitled, “Selective Elimination of Mitochondrial Mutations in the Germline by Genome Editing”. The authors report success using TALEN-based gene editing or mitochondrial-direct restriction enzyme (mito-ApaLI) to reduce the burden of mutant mitochondrial DNA (mtDNA). Their work was done primarily in mice, but also using chimeras made with murine oocytes fused with human cells bearing …Read More