A team of scientists led by Shoukhrat Mitalipov at Oregon Health and Science University announced this week that they used a technology known as CRISPR to edit sections of the human genome, performing the procedure on embryonic humans. It can lead to heart failure and sudden death in apparently healthy people.
Genetic engineering has always been a sensitive issue, but the scientists from Oregon Health and Science University in Portland said they believe they have broken new ground in the prevention of inherited disease by correcting deficient genes. "Consent is particularly important when dealing with very vulnerable research subjects, and human embryos are among the most vulnerable of God's creatures".
The study makes progress toward using gene editing to prevent genetic diseases, but there's still has a long way to go before clinical testing can begin, says Janet Rossant, a developmental biologist at the Hospital for Sick Children and the University of Toronto.
The experiments were privately funded; US tax dollars aren't allowed for embryo research. This was the first time such a trial was conducted in the United States, where the scientists erased an inherited heart defect from the embryos, a paper in the journal Nature said.
The process was tested on 18 lab-created embryos using sperm from the male donor and eggs donated by 12 healthy young women, the study said.
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.
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Still, the technology was not 100 percent successful. At the same time, the eggs were injected with gene editing tools. Then, the cut is spontaneously repaired by the cell with different mechanisms: one repairs the DNA without leaving any trace, while the other introduces some unwanted insertions or deletions of a few base pairs near the cutting site.
More than 10,000 disorders have been linked to just a single genetic error, and as the researchers continue with their work, their next target is BRCA, a gene associated with breast cancer growth. "Moreover, if such embryos were to grow up, as will doubtless occur in the future, there are likely to be unintended effects from modifying their genes", Fr. Pacholczyk continued.
Furthermore, to be ethical, any applications or experiments utilizing CRISPR or other gene editing technology can not use any other methods in its process which are themselves intrinsically immoral, Fr.Pacholczyk said. It is directed to a specific location in the DNA and performs a cut-and-paste function, not unlike word-processing software.
But the new embryo experiments were striking for both their efficacy and a lack of adverse events like mutations in other parts of the embryos' genomes. Embryos can be genetically screened before they are implanted during in vitro fertilization. While research like this is already occurring in China and Great Britain, this is the first time scientists in the US have edited an embryo.
Agus also noted that in the experiments, the technique proved to be very precise in targeting the defective gene, leaving the rest of the embryo's DNA "untouched". But the finding could be good news for those concerned about designer babies, because embryos may reject attempts to add new traits.