Germline Editing using CRISPR-Cas9 - Totipotent Cell Research

Dr.Yorgos Nikas/SPL
Yesterday the first published paper was released detailing the preliminary scientific attempts at modifiying the germline of a human fertilized egg - a zygote.

What was striking here wasn't so much the attempt being officially published but the storm it has caused in the scientific community.

The science is far from effective and needs a great deal of work before it is even possible to consider using in a clinical setting. That much was shown. There are many steps before any such work should be even contemplated, as guided by the ISSCR in their call for a clinical moritorium on proceding with any human translational work. I personally believe that position will be respected given the laws in place that govern such human germline science. If there is a precedent the ban on attempts to pursue human cloning has shown to be effective.

What is curious here in respect to this work, undertaken in China, on germline editing is that it has quickly followed the cautionary notices by the scientific community leadership that the science was being practiced in human germline cells. This was and is a signal that there is much more going on behind the scenes than we know and that the nature of such developments isn't necessarily coordinated or managed in any practical manner. To some that is the issue perhaps...

The explosion of interest and experimental use of gene editing technology has opened up a proverbial Pandora's box of issues that have yet to be addressed collectively in a meaningful manner. But as these things go it's not always possible to steer scientific advances as the peer review process is built to open up the knowledge gates to replicability and improvement - hence the strength in it's design. Coordinated stakeholder dialogue is urgently needed and initiatives established.

Man's curiosity and pursuit of knowledge has driven momumental changes in our own lifetimes - that is accelerating and those that wish to somehow control the speed of which it is happening may find themselves catagorized as part of the old guard and not hip to the trending interwoven nature of the instant always on tech culture of today and tomorrow.

The ever expanding numbers of brains educated to think of new solutions to existing problems with ubiquitious tools made easy will allow discovery and experimentation to flourish. No one should be surprised when this momentum spawns innovation - especially in the new frontier of biological system design.

My comments on Paul Knoepfler's Blog about the natural reservations of the developments are below:

"Thx Paul for the viewpoint. 

I believe you´re correct to have reservations about the unknown steps on the discovery path to gene line editing. However, I do believe it's important to participate in order to have any chance to mold the outcome. As we have just learned the science is indeed moving ahead, like it or not. I don't believe debating the merits of the possibilities does justice to the technology - at least to me that doesn't seem productive at this stage. 

Some may not wish to go down this discovery path but as many have pointed out - it's not something anyone or even a group(s) can put a halt to, it seems. 

I would like to see the West actively engaged in an initiative to put a scientific team of institutional investigators together to spearhead a collaberative international effort to lead coordinated research into geneline editing - in all it's forms (there are many of course research methods to explore). This way the open nature of such an initiative would be beneficial to all stakeholders and those on the ground can draw down from this central resource.

Attention grabbing science papers as a methodology to present the public the work being done adhoc doesn't strike me as proactively getting ahead of the issues... 

Also, isn't it possible to establish a seperate arm's length oversight department, in an existing organizational structure? This would officially sanction & coordinate the independent local discovery groups to inform and assist the lab work & validate data collection for the entire space. Or are we looking at global independent action, reaction, applied science and IP castle building for human elitism?"

Belmonte paper ref

Further on the issue of gene editing, today Juan Carlos Izpisua Belmonte’s group published a paper in Cell that suggests a possible route to modify mutated mitochondrial for disease elimination using an alternative to the above "CRISPR-Cas9" technology but similar in puprose "TALEN" system.

The sum of these parts and the overall emerging landscape of profound medical advancements is a net positive IMO, while there remains a need to establish a collective approach to coordinating the possibilities for Common Good.