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New Hope for Infertility and Healthy Babies as U.K. Approves Gene Editing Research on Human Embryos

Better understanding of the earliest stages of human development and the opportunity to improve fertility treatment and prevent miscarriages are goals of the British researcher who just received permission from United Kingdom (U.K.) regulators to use a powerful new genome editing technique on human embryos in the lab.
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Better understanding of the earliest stages of human development and the opportunity to improve fertility treatment and prevent miscarriages are goals of the British researcher who just received permission from United Kingdom (U.K.) regulators to use a powerful new genome editing technique on human embryos in the lab.

Dr. Kathy Niakan of London's Francis Crick Institute will be using the new gene editing technique, known as Crispr or Crispr-Cas9, which allows researchers to perform a sort of cut and paste with DNA. While the new research is not expected to change medical treatment immediately, the knowledge gained by using this new technique may prove helpful in treating different types of infertility, since many fertilized eggs fail before they reach the blastocyst stage when embryos are ready for implantation.

Many other researchers are eager to try the technique, too, as it's fast, easy to use, precise, and relatively inexpensive. It's also different than other gene therapies long-used on adults as this technique could change the genes of human eggs, sperm and early embryos, creating modifications that would be inherited by subsequent children.

The mechanisms being investigated by Dr. Niakan and her colleagues "are crucial in ensuring healthy, normal development and implantation" and could help doctors refine fertility treatments, according to Peter Braude, a retired professor of obstetrics and gynecology at King's College London.

The new gene editing research on embryos not only holds promise for couples facing infertility but may also help those with a family history of disease with a known genetic link such as muscular dystrophy and sickle cell disease. Now, such couples can have their embryos screened so that only healthy ones are implanted. This research could actually lead to editing the genes to make the embryos disease-free.

The permission granted to Dr. Niakan and her colleagues by the U.K.'s Human Fertilization and Embryology Authority represents the first time a country's government has approved use of this technique. The decision does not violate the current global voluntary moratorium on human DNA changes because the altered embryos will not be used to create babies. Instead of implantation, the embryos will be allowed to expire at seven days when they reach the blastocyst stage.

While countries may not require explicit government permission to conduct embryonic gene editing and the U.S. has no specific prohibition, there is a ban here on the use of federal funds to support research if a human embryo is destroyed in the process. The ban does not apply to privately-funded research.

Given differences in government attitudes towards research with human embryos, it is not surprising that British researchers have pioneered many of the advances in reproductive medicine once considered breakthroughs that are now are widely used. This includes the world's first so-called test-tube baby, embryonic stem cells (in mice, then adapted for humans) and mitochondrial replacement therapy. With formal approval to use this new technique, U.K. researchers are likely once again to be able to take the lead -- this time in better understanding of the earliest stages of human embryology and perhaps the implications for more effective infertility treatment as well.

The announcement that U.K. researchers may now begin using this new genetic editing technique on human embryos has attracted worldwide attention. While many are excited -- infertility specialists among them -- there is also criticism from other sectors including religious groups and some in the scientific community who worry about a "slippery slope" of designer babies.

For now, the sheer potential of this research to enhance our understanding of in-vitro fertilization (IVF) success rates by looking at the very earliest stage of human development should make us all happy at the idea of more, healthy babies for the families who want them...

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