An worldwide team of scientists has successfully edited of the genes of a viable human embryo, correcting a mutation that can cause the heart condition hypertrophic cardiomyopathy. It is the most common cause of sudden death in healthy, young athletes.
To achieve this major breakthrough, the researchers generated stem cells from a skin biopsy from a person with HCM and, using CRISPR, specifically targeted the MYBPC3 gene for fix.
An worldwide group of 11 organizations, including the American Society of Human Genetics and Britain's Wellcome Trust, on Wednesday issued a policy statement recommending against genome editing that culminates in human implantation and pregnancy, while supporting publicly funded research into its potential clinical applications. Crispr, although it appears to be very precisely targeted, can make errors analogous to a badly framed search-and-replace request, changing gene sequences that are far from the intended targets.
Federal agencies also ban funding of research that ultimately leads to the destruction of human embryos in research.
The researchers started with human embryos created from 12 healthy female donors and sperm from a male volunteer who carried the MYBOC3 gene.
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In December 2015, an global group of scientists convened by the us national Academy of sciences (NAS) in Washington had estimated that it would be "irresponsible" to use the technology of CRISPR to modify the embryo for therapeutic purposes as long as issues of safety and effectiveness have not been resolved.
The researchers found that 72.4 percent of the embryos, not 50 percent as would have been expected, were free of the errant gene. In the remaining 27.6% embryos, the cellular cut-repairing mechanism introduced some unwanted insertions or deletions near the cut.
The technique could be used through in vitro fertilization to cure thousands of diseases caused by mutations in single genes. Previously, scientists fertilized eggs and then added the CRISPR/Cas9 gene editor (top row).
The embryos were allowed to grow for five days for analysis.
In short, this experiment showed that it is potentially possible to correct a genetic disease in an embryo with a high chance of success.
"I for one believe, and this paper supports the view, that ultimately gene editing of human embryos can be made safe". It is directed to a specific location in the DNA and performs a cut-and-paste function, not unlike word-processing software. Though the researchers provided a replacement strand of DNA, the embryos instead took the mother's DNA as a guide to fix the MYBPC3 mutation. The work was consistent with recommendations issued this year by the National Academy of Sciences and the National Academy of Medicine joint panel on human genome editing.
Currently, the most reliable way of screening for such inherited defects is by using IVF, screening the resulting embryos for the mutation and transferring only those without the mutation for pregnancy. Any attempt to study germline editing in pregnant women would require permission from the Food and Drug Administration, which is now prohibited by Congress from reviewing any such request.