Genomic editing of viable human embryos using CRISPR/Cas9 could yield customized human embryonic stem cells, but ethical issues remain

The Human Fertility and Embryology Authority (HFEA) of the United Kingdom last week authorized a team led by Dr. Kathy Niakan at the Francis Crick Institute in London to edit the cells in viable human embryos, using the powerful CRISPR/Cas9 technology, in order to investigate the causes of miscarriage.  This is a controversial decision, since this is the first time that “germline” genomic editing has been approved by any government.

There are strict rules accompanying the HFEA’s permission to pursue this research:  the modified embryos cannot be implanted in a womb, and they must be destroyed after two weeks.

However, two weeks is long enough for the fertilized egg to develop into a blastocyst, the outer part of which, in the traditional course of events, will become the placenta and the inner part of which will become the embryo.  The inner cell mass can, alternatively, be re-purposed as human “embryonic stem cells,” (hESCs) by removing them from the blastocyst, a process that renders the blastocyst incapable of producing a fetus or a child.

Using CRISPR/Cas9 techniques to edit the genetic code of the first cells in the fertilized egg means that it would be possible to create human embryonic stem cells with specific and customized characteristics that are designed to treat a range of medical conditions or for further experimental purposes.

The upside of this process is the derivation of hESCs capable of treating disease or for further research.  The downsides include the inconvenience and discomfort for the women whose eggs are harvested, the expense of the process, and the ethical objections to the instrumental use of viable human embryos, which some critics consider to be the taking of a human life.

The ability to create customized embryonic stem cells through CRISPR/Cas9 genomic editing has not gone unnoticed by the scientists affiliated with the California Institute for Regenerative Medicine (CIRM), which met on February 4^th to discuss the issue of CRISPR/Cas9 gene modification in human embryos, as reported yesterday by Etopia News.

According to Kevin McCormack, Sr. Director of Public Communications and Patient Advocate Outreach at CIRM, at this meeting “the researchers did talk about cells harvested from blastocysts.”

CIRM identifies itself, on its website and generally, as “California’s Stem Cell Agency,” which makes sense, since it was created, through Proposition 71, the “California Stem Cell Research and Cures Initiative,” as an institution dedicated to pursuing breakthroughs in science that would enable the use of stem cells for a variety of therapeutic and research purposes.

The kind of germline genomic editing now authorized by the HFEA means that customized stem cells can probably now be created by using CRISPR/Cas9 to modify the first cells of the fertilized egg, letting the cell mass develop into a blastocyst and then harvesting the inner cells, which would then become human embryonic stem cells, possibly capable of treating a range of human diseases.

All this could be done within the two-week period authorized for experimentation on the embryo by the U.K.’s HFEA, or during a similar period as specified by CIRM.

The key ethical (and semantic) question then becomes “Does the rule established by the HFEA that the embryo must be “destroyed” after two weeks allow for the harvesting of the embryonic stem cells, a process that “destroys” the blastocyst, as required under the HFEA’s rules?”

Federal law prohibits the use of federal funds for human embryonic stem cell research.  This is why California created CIRM in the first place.  Current law in the U.S. neither authorizes nor prohibits CRISPR/Cas9 genomic editing of embryos, as long as federal funds are not used for the research.  CIRM was set up to investigate the use of human stem cells to treat disease.  CRISPR/Cas9, discovered and developed after CIRM’s establishment, now gives scientists a powerful new tool capable of creating customized hESCs.

Will California, under the leadership of CIRM, pioneer this new process, or will ethical objections block its development?  Such objections were raised during and after the campaign to pass Proposition 71 itself.  

Does CIRM have the authority to authorize such CRISPR/Cas9 research on viable human embryos in California?  Will it award grants to the state’s scientists to conduct this kind of research and the treatments derived from it?

Keep reading Etopia News to follow this issue as it evolves and find out.

Assemblymember Jim Cooper’s office explains what it wants Apple and Google to do about encryption

California Assemblymember Jim Cooper is the author of a bill, AB 1681, that would prohibit the manufacturing of smartphones capable of letting their users encrypt their “data at rest” beyond the reach of law enforcement, even when the authorities have a proper warrant to see the information.

Skyler Wonnacott, Assemblymember Cooper’s Communications Director, spoke today with Etopia News and had some things to say about the bill.

He accused Apple and Google of “putting profit before people” by offering an operating system (iOS8 and Android 6.0) that puts the last word on encrypted data in the hands of the users and the passcode known only to them, beyond the reach of the operating system’s creators and, hence, beyond the reach of any law enforcement or surveillance agency that seeks access to it by sending the makers of the software the smartphone, specifications of the data to be “pulled” (decrypted), and the legally-valid warrant giving them permission to access that data.

Speaking about the software versions that provide default total-disk encryption, he said that Assemblymember Cooper wants Apple and Google to “roll it back or create a new operating system that would allow them to decrypt the user’s data.”

Wonnacott emphasized that such retrieval of information from a person’s smartphone would only take place “when a search warrant has been issued.”  He said that he was “totally fine with sending them to the manufacturer” to have specified data “pulled” from the device, rather than giving law enforcement itself the ability to read the encrypted data.

Asked about any response to this legislation from Apple or Google, he said that he had so far heard “not a peep out of Apple, not a peep out of Google.”  He added that “they have not reached out to us.  We’re open to talk.”

As for the status of the bill itself, he said that it had not yet been referred to committee. Roy Sianez, Legislative Director for Assemblymember Cooper, said he thought it would probably be assigned to the Privacy and Consumer Protection Committee or perhaps to the Judiciary Committee, or maybe even both.

Wonnacott said he thought the bill would probably get its committee assignment in “mid-March.”

CIRM discusses human embryo editing with CRISPR and decides to discuss it further

According to its website:

“California's Stem Cell Agency was created in 2004 when 59% of California voters approved Proposition 71: the California Stem Cell Research and Cures Initiative. That initiative created the California Institute for Regenerative Medicine (CIRM) to fund stem cell research in the state. In addition to creating the agency, Prop 71 created a 29-member governing Board composed of researchers, business leaders and patient advocates.”

Somewhere along the way, CIRM had the good sense to hire Kevin McCormack as its Sr. Director of Public Communications and Patient Advocate Outreach.  

Yesterday, the CIRM’s Standards Working Group (SWG) held a workshop to talk about the use of CRISPR to edit human embryos.  Unfortunately, the telephonic audio bridge that they had arranged to transmit the meeting to the public was of such low quality that few people could hear what was going on.

Fortunately, Kevin McCormack prepared a summary of that workshop meeting.  Here it is:

The meeting began with the hope that it would produce a strong, robust discussion of the issues surrounding the use of CRISPR to edit human embryos and to result in a series of recommendations that the Standards Working Group (SWG) could then forward to the CIRM Board on whether any changes needed to be made to our existing rules and regulations about funding such research.

It turned out to be a thoroughly fascinating day with some thought-provoking presentations and equally thought provoking questions from the audience, from scientists, social researchers and members of the public. 

It quickly became clear that the discussion was going to be even more robust than we imagined and the issues raised were too many and too complex for us to hope reaching any conclusions or producing any recommendations in one day. 

In the end it was decided that the most productive use of the day was not to limit the discussion at the workshop but to get those present to highlight the issues and questions that were most important and leave it to the SWG to then work through those and develop a series of recommendations that would eventually be presented to the Board.

The questions to be answered included but are not limited to:

1)   Do we need to reconsider the language used in getting informed consent from donors in light of the ability of CRISPR and other technologies to do things that we previously couldn’t easily do.

2)   Can we use CRISPR on previously donated materials/samples where general consent was given without knowing that these technologies could be available or can we only use it on biomaterials to be collected going forward.

3)   Clarify whether the language we use about genetic modification should also include mitochondrial DNA as well as nuclear DNA.

4)   The possibility that somatic cell gene editing may lead to inadvertent germ line editing

5)   How do we engage with patient advocates and other community groups such as the social justice and equity movements to get their input on these topics – do we need to do more outreach and education among the public or specific groups and try to get more input from them (after all we are a taxpayer created and funded organization so we clearly have some responsibility to the wider California community and not just to researchers and patients)

6)  As CIRM already funds human embryo research should we consider funding the use of CRISPR and other technologies that can modify the human embryo provided those embryos are not going to be implanted in a human uterus. 

This was a really detailed dive into a subject that is clearly getting a lot of scientific attention around the world and is no longer an abstract idea but is rapidly becoming a scientific reality. The next step is for a subgroup of the SWG to put together the key issues at stake here and place them in a framework for another discussion with the full SWG at some point in the future.

Once the SWG has reached consensus their recommendations will then go to the CIRM Board for its consideration.

I hope this captures the flavor and essence of what happened today. It really was a fascinating discussion and the issues raised, and their complexity, highlighted why so many different groups around the world are wrestling with the potential, and pitfalls, of this new technology. 

California gubernatorial candidate Gavin Newsom hasn’t taken a position on CRISPR-Cas9 embryo editing yet; CIRM meets to discuss the issue

California Lt. Gov. Gavin Newsom is running for Governor of California in 2018.  As previously reported on Etopia News, he has not yet taken a position on AB 1681, a bill by California Assemblymember Jim Cooper that would prohibit the seemingly-unbreakable encryption enabled by iOS8 and Android 6.0 for smartphones.

He also hasn’t taken a position on the use of the powerful new CRISPR-Cas9 gene editing technology when it comes to using it to edit human embryos, as was just approved by the UK’s Human Fertilization & Embryology Authority (HFEA), for an experiment being conducted at the Francis Crick Institute, by Dr. Kathy Niakan and her team, to investigate early embryonic development.

In an e-mail sent today to Etopia News, Rhys Williams, his Chief of Staff, wrote:

“California’s Lt. Governor has not yet reviewed the UK’s HFEA decision. “

The California Institute for Regenerative Medicine, according to Kevin McCormack, its Sr. Director Public Communications & Patient Advocate Outreach, is today putting on a workshop by its Standards Working Group “that looks at that precise question.”

You can join this workshop’s audio conference at:  800-230-1951.  Use the confirmation number 385423.  The workshop is scheduled to continue until 4:00 pm, PST today.