NgAgo a-go-go: top 5 bullet points on upstart CRISPR challenger

NgAgoThe gene editing technology CRISPR has been arguably the top story in the biomedical world in the last two years, but going forward there is a CRISPR challenger in upstart gene editing technology NgAgo.

For more background on NgAgo and the key first published paper on its genetic modification characteristics see my post here. 

In the comments on that post and in discussions I’ve had with other researchers, some key points have crystalized on NgAgo versus CRISPR at this time. As a possible CRISPR challenger, how does NgAgo fare?

Broader possible applicability. The lack of a PAM site requirement for NgAgo means it is almost certain that for some specific gene editing applications, NgAgo will work and CRISPR won’t. Design of NgAgo guides seems to be a simpler matter too because no PAM is needed (more on guides below). In that first NgAgo paper they reported effective editing of 8 different genes with good efficiency so it’s unlikely there is a strongly required DNA sequence context needed for NgAgo. However, it is still formally possible that NgAgo in some contexts will have some kind of preference for certain DNA sequences.  Further study will help resolve this more concretely, but so far this is looking like a major plus for NgAgo.

DNA guides should be a lot easier. The use of DNA-based guides will make gene editing easier as opposed to RNA-based guides. At the very least you eliminate a cloning step and you can just order oligos, which you can phosphorylate in your lab to use as guides by transfection.

Continue reading

Anti-GMO poster in Switzerland invokes human GMOs

Over on Twitter Magdalena Plotczyk (@MPlotczyk) posted a striking photo of an anti-GMO poster from Lausanne, Switzerland. The top part of the poster translates as, “‘After GMO corn, GMO children?'”

GMO Corn, GMO People

Magdalena Plotczyk

As readers of this blog know, I do have concerns about the eventual production of genetically modified people using rapidly evolving genetic modification/gene editing technology such as CRISPR.

In fact, I’ve written an entire new book on this, GMO Sapiens. I hope you’ll read it. However, as you’ll see in the book, I’m not so concerned about GMO plants and foods in a general sense. I also do not see that GMO people or as I refer to them in the book, GMO sapiens, directly follow from GMOs in the plant world.

I think the bottom part of the poster translates roughly as, “No to unlimited reproductive medicine”.

Here’s the tweet.

Haruko Obokata (小保方 晴子) website posts dubious STAP cell validation data

Haruko Obokata is most well-known for her role as first author of the now retracted two STAP cell Nature papers. These manuscripts claimed to have made pluripotent and even totipotent stem cells simply by stressing cells out with acid treatment or in other ways. Nobody else could get this method to work to create the so-called STAP cells.

It was an all around scientific disaster and I don’t know anyone who believes that STAP cells are real, but Obokata and another one of her mentors, Dr. Charles Vacanti have still at times indicated their belief in STAP.

STAP cell Obokata 2016

Screenshot from STAP Hope website

Obokata appears to have launched a new website at the end of March of this year and there was a sense that this site along with her memoir-like book would together tell her side of the story plus might continue to push the notion that STAP is real. Update: it is formally possible that Obokata is not running this website so I have made a few change to this post.

Continue reading

Huge clinical trial patient fees allowed by FDA at times, details often secret

stem cell monopolyI am often critical of for-profit stem cell clinics on this blog for numerous reasons. For instance, one thing that concerns me greatly about these clinics is that they charge patients to get experimental “treatments” that have not been proven to be safe or effective.

But as some patients have pointed out to me over the years, certain FDA-approved stem cell clinical trials also charge patients to receive unproven stem cell therapies.

Should someone (even if that someone is an academic clinical researcher) be able to charge a patient a large access fee to be in a clinical trial in which that patient will be subject to an experimental therapy that could ultimately be proven unsafe and ineffective?

I have serious doubts about this.stem cell monopoly quote

You might be surprised to learn that the FDA in some instances allows patients to be charged to be in trials including stem cell trials and others. Those running the trials can request permission from the FDA to charge patients. The agency then has to evaluate the request. The FDA has a draft guidance on this, but frankly it’s difficult to learn that much from that document in terms of details in how this plays out.

Continue reading

TGIF Science: funding, CRISPR v. NgAgo, secrets, Zika, & more

Some stuff on my mind for our TGIF Science this week.

Research Funding Ups: NIH. Is it my imagination or is NIH funding slightly improving? This is the overall vibe I’m hearing from the trenches.

TGIF science dart board

Modified Wikipedia image

Research Funding Ups and Downs: CIRM.  CIRM funded some basic research to the tune of a total of $4 million, which is great. On a personal level, just wish my “great idea” had fared better in review there. Kind of a downer, but after a while you get a really thick skin.

I’m still trying to decide what I think of CIRM 2.0’s grant review, especially their “positive” pre-review where there is no scoring or comments. You either make it or are triaged with no explanation or score given to applications. No sour grapes here from me on this particular DISC1 CIRM grant review, but even though my proposal made the cut to get fully reviewed, the actual review was too short, just a handful of vague sentences in total. That’s not helpful.

Secret genome meeting? I’m still scratching my head over that “secret” meeting on a synthetic human genome over at Harvard. They closed the door on the public, the press, etc. The reason given was that a journal had embargoed the heck out of ideas that would be presented at the meeting or something like that. 

CRISPR obsolete soon? George Church was quoted that CRISPR will be obsolete soon because of synthesizing entire genomes from scratch instead (see secret meeting entry above). Then of course there’s the upstart NgAgo that could be simpler and with broader applicability than CRISPR. What do you think of CRISPR versus NgAgo?

My sense is that talk of CRISPR obsolescence is premature kind of like Mark Twain’s quote that reports of his death were exaggerated.

Continue reading