CRISPR Cas9 Corrects MYBPC3 Mutation in Human Embryos
“Using CRISPR Cas9, they successfully corrected the MYBPC3 mutation – which causes hypertrophic cardiomyopathy, the leading cause of sudden cardiac death in young athletes – in viable human embryos”
Summary
Researchers have used CRISPR‑Cas9 to correct the MYBPC3 mutation in pre‑implantation human embryos, demonstrating efficient gene editing and reduction of the mutant allele. However, the studies did not show that the edited embryos were viable to term or resulted in live births, so the claim of successful correction in viable embryos is overstated.
Sources 60 searched
- Gene Editing of Human Embryos with CRISPR/Cas9: Great Promise Coupled with Important Caveats - PMC
Several research groups in China ... Ma et al.1 reported that 42 (72.4%) out of 58 human embryos fertilized with sperm carrying the heterozygous MYBPC3 mutation carried two copies of the non-mutated gene copy after CRISPR/Cas9-treatment, as opposed to 47.4% in the control, ...
- Correction of a pathogenic gene mutation in human embryos | Nature
Here we describe the correction ... embryos with precise CRISPR–Cas9-based targeting accuracy and high homology-directed repair efficiency by activating an endogenous, germline-specific DNA repair response....
- ‘Biotech Barbie’ says the time has come to consider CRISPR babies. Do scientists agree?
A company’s plan to edit the genomes of human embryos worries some researchers — but it might reflect the changing attitudes towards the controversial approach. A company’s plan to edit the genomes of human embryos worries some researchers — but it might reflect the changing attitudes ...
- Correction of a pathogenic gene mutation in human embryos - PubMed
Here we describe the correction ... embryos with precise CRISPR-Cas9-based targeting accuracy and high homology-directed repair efficiency by activating an endogenous, germline-specific DNA repair response....
- Using gene editing technology to repair a pathogenic mutation in human embryos | Research Communities by Springer Nature
Correction of a MYBPC3 mutation causing hypertrophic cardiomyopathy by CRISPR-Cas9 in preimplantation human embryos by injection of unfertilized oocytes.
- CRISPR/Cas9 and reproductive failure: applications, ethical challenges, and future perspectives in human germline genome editing - ScienceDirect
This review critically examines current applications of CRISPR/Cas9 in reproductive biology, including disease modeling in animal systems, editing of spermatogonial stem cells (SSCs), manipulation of oocytes and zygotes, and proof-of-concept studies in human embryos.
- Gene-editing human embryos to prevent disease : Shots - Health News : NPR
Harrington envisions using newer and hopefully safer gene-editing techniques, such as "base editing," to modify embryos to make babies. He said his focus too would be on preventing diseases. The Chinese scientist used the gene-editing technique known as CRISPR, which allows scientists to make very precise changes in DNA much more easily than ever before but can cause potentially dangerous random mutations.
- The CRISPR Babies | Science History Institute
At this point, not a single correction. Our report has stood the test of time. Alexis Pedrick: At this point, the public was aware of He Jiankui’s claims, that there were seven couples who went through embryo editing in IVF, and one of the pregnancies had resulted in the birth of twins.
- Human germline engineering - Wikipedia
The claims of this study were however pushed back on by critics who argued the evidence was unpersuasive. A June 2018 study researchers reported a potential link for edited cells having increased cancerous potential. The study reported that CRISPR/Cas9 induced DNA damage response and stopped ...
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