A groundbreaking gene editing technique just had a massive success, and could represent a big step forward to solving a host of deadly diseases.
In what could prove to be an incredible breakthrough, scientists have found a way to modify DNA in human embryos without introducing human mutations, which could pave the way for future gene editing practices that could eliminate a wide range of deadly diseases from cancer to diabetes. An international team of scientists published their findings in the journal Nature describing the CRISPR technique that could use to combat genetic diseases.
The CRISPR gene-editing technique was used to correct a genetic defect that results in a heart disorder in young people in two thirds of embryos that were tested, and all without causing a dangerous mutation.
It’s an astounding discovery that could represent a gigantic leap forward in medicine, although it still has a long road to go to pass further tests and trials to prove that it is safe and effective in humans. But it is an exciting development that could have tremendous benefits for mankind.
“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. The efficiency, accuracy and safety of the approach presented suggest that it has potential to be used for the correction of heritable mutations in human embryos by complementing preimplantation genetic diagnosis.”