gene editing


September 2017 with Nathan and Laura: Venter Blunder, RNAi Returns, and Monthly Science Moments

To honor Laura's pentametric thirst,

We write the summary today in verse.

 

Was it a quake that had no epicenter,

That silly paper out by J. Craig Venter?

 

And after years of silencing the market

Has RNAi at last knocked out its target?

 

Then Nathan gives to yuppies devil's choice.

Which one libs: gluten dough or GMOs?

 

August 2017 Review with Nathan and Laura: CAR-T Cashes In, Embryos Edited in US, and the Invitae Incident

Back from summer vacation, Nathan and Laura are smoking hot as they look back over some exciting headlines.

The summer boiled over with plenty to talk about, but it was just this week that delivered most of the news for our discussion today. Novartis’ gene therapy based on CAR-T technology was approved Wednesday, making it the first gene therapy to be approved ever in the US. Analysts will be trying to figure out how high high is when it comes to the price tag, but Nathan and Laura explain why this therapy is a big deal for patients.

As for the first gene editing of embryos in the US that happened earlier in August? Nathan says, yes, it’s a first, but the big story is how "strikingly reliable the CRISPR edit is in germline vs the rest of the body."

Finally, we heard a few days ago that genetic testing provider, Invitae (recently featured here on the program) had sent out a large batch of false negative tests. Laura, a genetic counselor, says that in the absence of FDA regulation the system is operating on trust.

“And I want to say,” she adds, “ I trust Invitae. They’re a good lab, and I think they’re handling this well.”

 

The First In-Human Gene Editing Trial in the U.S. - And It’s Not with CRISPR

The challenge for the first ever in-human gene editing trial, according to today’s guest, is with the delivery to the body.

“At the moment, the easiest place to deliver your gene or genome editing is to the liver, using AAV which are viruses that seek out and go to the liver cells," says Sandy Macrae, the CEO of Sangamo Therapeutics.

Sangamo is known for two things: They have pioneered the commercialization of an older gene editing technology called Zinc Fingers. And they have done a lot of work in the area of HIV.

Today, Sangamo is enrolling patients in a new trial which they say will be the first "in-vivo" trial using their Zinc Fingers for patients with hemophilia B, Hunter syndrome, and Hurler syndrome. The former gene editing work with the T cells of HIV patients, Sandy says, was done “ex-vivo”, or outside the body.

So why is Sangamo still using Zinc Fingers in the age of CRISPR? Sandy says that the older technology is much better developed for medical applications and is safer. The company has been able to get their off target effects to below the level of detection.

“When I was doing my postdoc, I would have used CRISPR. It’s better if you’re just wanting an easy experiment that isn’t about making a medicine but just getting a quick answer,” he says.

Because Sangamo has been the sole commercial developer of Zinc Fingers with not a lot of intellectual property dispute, the technology didn’t make the big PR splash that CRISPR has—nor, at the same time, did it generate all the fear.

We finish the interview with a question about what was the result of all Sangamo's work in HIV over the years.

George Church on What Comes After CRISPR

George Church joins us today. He’s the Robert Winthrop Professor of . . . . well, he’s George Church. And he confirms that, yes, a movie called “Woolly" is being made about his lab. In the next breath, he reminds us (and himself?) that less than 1% of his press is about the woolly mammoth.

George was honored this year as one of Time Magazine's "Time 100 List", and we’re curious about what topics of conversation come up when he’s with non-scientists. What does he think of Trump (or what he calls “the new politics”)? Is he tempted to join the wave of scientists running for office?

“In China, about 80% of their top government officials have a degree in science or engineering, while in the United States it's closer to 1%,” he deadpans.

We cover some scientific ground today as well. George says CRISPR has been overhyped as the step between Zinc Fingers/Talens and the next gene editing tool. So what is the next CRISPR? And what ever came of his 3-D sequencing?

Enjoy.

Is CRISPR Controversy Science or Spin? June 2017 Review with Nathan and Laura

It’s the end of the month--and the half year mark--so we open up today's monthly discussion with Nathan and Laura to include some of the headlines we’ve missed this year.

Last month a paper was published warning about the off target effects when using CRISPR. Laura and Nathan agree the kerfuffle which exploded into this month was more about Wall Street than adding anything new to science.

Remember the technology we used before CRISPR? Sangamo Biosciences launched the first ever “in vivo” (in human) trial for gene editing using Zinc Finger technology.

Also last month, the FDA approved a drug based on the genetic makeup of a tumor and not its location in the body. Nathan says, “Nyeh . . . And? Doctors have been doing it off label for years.” But Laura thinks it's more that just a symbolic gesture.

She also picked for her paper of the month this new study out by Robert Green’s group asking whether the benefits outweigh the risks for whole genome sequencing in healthy individuals.

"It’s preliminary, but it’s not PR. This is one of the really important questions for genomics in our time," says Laura.

Nathan highlights a paper propounding a new model for biology, the omnigenic model. Find out what that is in another fun and informative look back on the genomics headlines with Nathan Pearson and Laura Hercher.

Over $1 Billion Invested this Past Year: Synthetic Biology in 2017 with John Cumbers

What does it take to make it in synthetic biology in 2017?

Working as a bio engineer at NASA, John Cumbers founded SynBioBeta, the primary “activity hub” for the synthetic biology community. SynBioBeta will be putting on their sixth conference this year in San Francisco, along with conferences in London and Singapore. The young industry has seen a flourish of startups working on new genome engineering tools and a dizzying array of applications that include synthetic animal meat and synthetic human skin. Last month John partnered with Data Collective to launch a new seed stage fund for this space.

John is not only interested in startups. Currently writing a book with the working title, “What’s Your Bio Strategy?”, he is provoking existing companies to consider using biology as technology.

“The book is designed to be something we could take into [Apple CEO] Tim Cook’s office, and ask him what’s your bio strategy. And he says, 'I don’t have a bio strategy.' So you put the book on his desk and say give me a call if we can help you to develop one.”

When we first talked with John, he was heading up a program at NASA to develop building materials for use on Mars. Five years later, is John's number one goal in life still to settle the solar system?

In-Situ Sequencing, CRISPR Patents, and Racist Milk Drinkers: February 2017 with Nathan and Laura

Commentators Nathan Pearson and Laura Hercher join us to look back on February’s genomics headlines.

Beginning this time with science, Nathan says we should be expecting great things from new in-situ sequencing. Laura found it encouraging that the National Academy of Sciences shifted to be more in support of genome editing. Theral asks what life forms are left to sequence for the Earth BioGenome Project?

Then it’s back to politics. Are the departure of Liz Mansfield from the FDA and Matt Might from the White House the beginning of a brain drain from government agencies in the new administration? We finish with some stories about racism that might fit under the heading “family genomics and black history month."

Gene and Tonic: A 2016 Timeline

 

Journalists listen to others telling them what actually happened all year long.  But for this one week at the first of the year, we like to make up our own stuff.

January - The general mood at the annual J.P. Morgan Healthcare conference in San Francisco is one of relief.  

“Last month the FBI caught the lead mastermind behind the pharma industry’s high drug prices, and he’ll be brought to justice,” says the CEO of a pharma giant to a room full of investors and journalists at the historic St. Francis Hotel.  “Problem solved.”

I Prefer My Bacon Crispy: Why I Don’t Think CRISPR is Really That Big of a Deal

With all the recent news around CRISPR my reaction is “meh” (coincidentally, the same reaction I’m having to the current US presidential election noise). We are a wee bit early for both.

Is Jurassic Park – and now Jurassic World real? No.

Gene and Tonic: Sexism in Science, How to Spend an NIH Budget Increase, How Not to Spend It

Janitors have had a terribly busy time this week cleaning up all those jaws that were dropped on floors of research labs everywhere around the country.

Have you heard about this latest sexism scandal?

Two female co-authors of a scientific paper submitted their work to PLOS -- you know, the open access journal.   You won’t believe what they heard back from the lone peer reviewer.  They were told to go find “one or two male biologists” to be co-authors on the paper to increase its chances of being published.

Ouch!!!  That hurts.  Not only the co-authors but the rest of us.

Well, hold on, it gets worse.  This chauvinist reviewer went on to say that “it might well be that on average men publish in better journals . . . perhaps simply because men, perhaps, on average, work more hours per week than women, due to marginally better health and stamina.”

What, a marginal ouch?  Better health and stamina?

Then the two female co-authors decided to stop playing that video game, got their scarves, and went across the street to a cafe and ordered each a double latte.

Right?  I mean, what’s the name of the video game, Doing Science Circa 1850?

"No," the lead female author says sitting down to her double latte.  "The game is called, Anti-Civilization;  Hang Out with a Primitive Tribe in Africa."

Now, last week we reported how former Congressman Newt Gingrich is calling for the  doubling of the NIH Budget.  Well this week, the bipartisan 21st Century Cures Initiative jumped in the game, calling for an increase to the NIH budget.  $1.5 billion per year over the next five years.  Well, we still like Newt’s suggestion.  It’s bold.  And let’s be positive.  Let’s say we get the budget doubled.  Now we got the problem of spending it.  Right?  Be careful what you wish for.

Since we’re absolutely sure that the NIH will come to Mendelspod for suggestions on how to spend the increase, we thought we should at least start getting prepared.

So we went online and posted a chat asking for suggestions from researchers how to spend the additional funds. 

Would you like to hear a few of the responses?

Garbage In, Garbage Out - obviously the commentors are using pseudonyms -- from Phoenix, AZ, writes: “Write off half of it to waste. Because that’s the way it is. Over 50% of scientific research is non-reproducible.”

OK.  We’re being taken seriously here.

Live to One Thousand from Cambridge, England, writes:  “Spend all of the additional funding on aging research.  We’ve tried the sniper method.  Let’s just move in the troops.”

Wow, this is a serious chat.  But hold on, a third person, Don’t Leave us Behind, out of San Diego, CA, writes:

Are you sure, Live to One Thousand?  Aging research?  I think we should take the additional $30 billion and fund Alzheimer’s research.  You see, Nature is now asking us, are you sure you want to live longer?

Oh, and there’s one more here that just came in.  It’s the author of the book called, From Buddhism to Big Dataism: Keeping up with the Newest Religions.  And this author writes:  "You might as well write the check now, NIH.  Just make it out to the newest God on the Block, Big Data.  

These online chats.  They’re just too serious.

Now with all this talk of increasing the research budget, Francis Collins, the Director at NIH, immediately put out a notice about what he won’t fund.  OK, he’s showing congress that he’s a good accountant.  So what will the NIH not fund?  Editing the human germline in embryos.

Now this is the only one he’s announced so far, but we heard that there are more.  Did you want to hear about a couple?

OK, here’s one.  This is something that the NIH will just not fund, no matter what.  The proposal came in to do brain scans of all the presidential contenders and make the data openly available online for all the voters to see.

And here’s a whole category of projects to study why people are gay.   What’s wrong with that?  It turns out a Supreme Court Justice told the NIH that such studies are a gross overreach of the executive branch.  That the direction of American society should not be up to scientists, but instead up to nine aging lawyers.  "Besides," this justice said to the NIH, "we have the better costumes.  White lab coats? Ha!"

 


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