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General Category => Off the Record => Topic started by: viper37 on August 07, 2017, 07:34:48 AM

Title: Correction of a pathogenic gene mutation in human embryos
Post by: viper37 on August 07, 2017, 07:34:48 AM
Link to Nature (https://www.nature.com/nature/journal/vaop/ncurrent/full/nature23305.html)

Link to La Presse (french) (http://www.lapresse.ca/le-soleil/actualites/science/201708/06/01-5122466-un-prof-de-lul-derriere-la-premiere-correction-dun-embryon-humain.php)


QuoteGenome editing has potential for the targeted correction of germline mutations. 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. However, much remains to be considered before clinical applications, including the reproducibility of the technique with other heterozygous mutations.


Coordinators, Gatica, Khan, all this could soon be a reality...

Ok, lots of work before we get there, but it's still a pretty amazing breakthrough.
Title: Re: Correction of a pathogenic gene mutation in human embryos
Post by: jimmy olsen on August 07, 2017, 07:42:17 AM
Time for the clone wars! :w00t: