On Embryonic Gene Editing, California May Follow UK

By Lila Abassi — Feb 09, 2016
The California Institute of Regenerative Medicine, a state-run agency that funds stem cell research, is considering funding research on human embryos using a gene-editing technology known as CRISPR-Cas9. This could potentially allow for cures to be discovered for devastating inherited diseases.
gene editing via shutterstock gene editing via shutterstock

The United States government does not support funding for any research that involves genetic alteration of human embryos, or human germline cells (sperm and eggs), that can be inherited by offspring.

Meanwhile, the United Kingdom s Human Fertilisation and Embryology Authority, a regulatory body, recently made headlines by granting permission to researchers at the Francis Crick Institute to edit genomes of human embryos. The genes will be manipulated and observed up to seven days of cell division the blastocyst stage and then be destroyed.

Gene editing in human embryos is not illegal in the U.S., only that the government does not fund that type of research. That said, it doesn't mean that private or state agencies are barred from doing so. The California Institute of Regenerative Medicine, a state run organization, funds research on stem cells and is considering taking the lead in financing the efforts to conduct experiments on human embryos and germ line cells.

There is a worldwide moratorium on gene-editing techniques that are heritable with the chief concern being that this technology might be inappropriately used to create designer babies.

Edward Lanphier, chairman of the Alliance for Regenerative Medicine in Washington DC, and four colleagues, call on scientists to agree not to modify human embryos even for research.

Such research could be exploited for non-therapeutic modifications. We are concerned that a public outcry about such an ethical breach could hinder a promising area of therapeutic development, said Lanphier and his co-authors, in a statement published in Nature.

Similarly, in an item published in Science, geneticist Dana Carroll of the University of Utah in Salt Lake City, says there should be discussions of the safety and ethics of using editing techniques on human embryos.

Germ line genome alterations are permanent and heritable," Carroll said, "so very, very careful consideration needs to be taken in advance of such applications.

What makes this technology a possibilty? There is a real potential of being able to correct genetic disorders through the development of possibly the most revolutionary scientific achievement of the millennia CRISPR-Cas9.

Crisprs (clustered regularly interspaced short palindromic repeats) are sections of DNA, while Cas9 (Crispr-associated protein 9) is an enzyme (see in this video). This technique adapts a natural defense mechanism that bacteria use to fight off invading viruses (by identifying and attacking viral DNA) to identify and "edit" DNA targeted by researchers, and it allows precise gene-editing to be radically less expensive and much more accurate than ever before.

Should we allow fears of a Brave New World to guide our decisions with respect to the potential of curing countless diseases? Until all safety and efficacy issues have been resolved, it would be extraordinarily irresponsible to introduce any genetic modification into the human germline. Gene editing, should, without a doubt be studied ad nauseum, and I trust that the scientific process will allow us to eventually ensure that it will be safe and effective. But until that time, how do we really know that a change in one area of our genome will not have unintended downstream effects?

Undoubtedly, this area of research has and will continue to provoke significant heated debates on both sides of the ethical fence.

The CRISPR-Cas9 technology has opened the doors of unlimited potential but precautionary measures need to stay at the forefront of any scientific research. We are not so far removed from a very ugly chapter in human history which legitimizes our fear of eugenics.

According to Tanya Lewis, in an article in Business Insider, These enhancements may also be unevenly distributed among the population, leading to a society of genetic haves and have-nots, and the inequity of the distribution and access to genetic therapy is the most plausible of all fears.