A dramatic new finding by scientists has found a way to fix genetic diseases in human embryos without introducing mutations.
Scientists have just achieved an astonishing feat, proving that a gene-editing technique can eliminate the risk of many serious genetic diseases while not introducing dangerous mutations as had been the case in the past. The paper, published in the journal Nature, was created by an international team of scientists trying to find a way to edit DNA with the CRISPR technique in a way that would prevent many genetic diseases from ever materializing.
They used the gene-editing technique to correct a genetic defect that can result in a heart disorder that can prove dangerous for young people, and they were remarkably successful in two thirds of embryos tested, and they didn’t introduce a mutation that would prove harmful.
It’s a groundbreaking discovery that, if confirmed with future tests and trials to be safe and effective, could have a massive impact on the medical community for many years to come.
“Genome editing has potential for the targeted correction of germline mutations,” the abstract states. “Here we describe the correction of the heterozygous MYBPC3 mutation in human preimplantation embryos with precise CRISPR–Cas9-based targeting accuracy and high homology-directed repair efficiency by activating an endogenous, germline-specific DNA repair response. Induced double-strand breaks (DSBs) at the mutant paternal allele were predominantly repaired using the homologous wild-type maternal gene instead of a synthetic DNA template. By modulating the cell cycle stage at which the DSB was induced, we were able to avoid mosaicism in cleaving embryos and achieve a high yield of homozygous embryos carrying the wild-type MYBPC3 gene without evidence of off-target mutations.”
