Tag Archives: DNA

The Man Who Wrote the Book on CRISPR and What He Taught Me

Kevin Davies literally wrote the book on CRISPR, the revolutionary new gene editing technology that earned Jennifer Doudna and Emmanuelle Charpentier a Nobel Prize a few months ago. Today, I had the wonderful opportunity to attend a talk he gave on that superb book, Editing Humanity.

If you’re not familiar, CRISPR is a technology based on the immune systems of bacteria. Bacteria find a particular genetic sequence they recognize from past infections and cut the genes in order to protect themselves. Scientists have harnessed this primordial system to cut and splice genes.

In today’s talk, Davies highlighted how accessible this revolutionary new technology really is. The equipment is not expensive and many labs could potentially use it. This presents great opportunities but also very real risks of misapplication.

CRISPR has been used successfully to treat sickle cell anemia in early trials. Davies noted that it has also been used to treat progeria in mice, which might some day bring an end to this deadly disease that ages children before their time.

I even got the opportunity to ask Davies a question, and inquired which other applications of CRISPR excite him most. He mentioned possible applications for cystic fibrosis and cancer therapy. He also said that as a graduate student in genetics, the idea of precisely editing genes seemed like science fiction, but today is a reality. It amazed me to think of how much the field has evolved.

Another interesting tidbit from the talk: due to COVID, Doudna accepted her Nobel Prize in the backyard of her home in Berkeley! I found that image to be quite a beautiful one.

One great silver lining of COVID has been how much easier it’s become to attend talks like this! In the past, one might have had to be in Cambridge, MA to attend, but now it’s open to everyone. I hope we continue to offer a remote option for these discussions even once in person events are possible again.

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Photo: “File:Emmanuelle Charpentier and Jennifer Doudna.jpg” by For Emmanuelle Charpentier portrait, credit Bianca Fioretti of Hallbauer & Fioretti. For Jennifer Doudna portrait, credit User:Duncan.Hull and The Royal Society. is licensed under CC BY-SA 4.0

What I Learned From James Watson, Co-Discoverer of the Structure of DNA

We wish to suggest a structure for the salt deoxyribose nucleic acid (DNA). This structure has novel features which are of considerable biological interest.

So begins the famous Nature paper by James Watson and Francis Crick, who discovered the structure of DNA in 1953. Their discovery revolutionized biology and won them both the Nobel Prize.

I just finished reading Watson’s book The Double Helix, and as a layman I found the inside view of the process of scientific discovery intriguing. What struck me most was the importance of collaboration.

We are used to thinking of scientists as lone geniuses hunched over a lab bench until they exclaim “Eureka!” But Watson’s book makes clear how important the many scientists surrounding him at the University of Cambridge were to his discovery. He repeatedly checks his findings with others more experienced than he in a particular area, like structural chemistry. And without the long conversations with Crick, the discovery would never have happened in the first place.

Being in the right environment was so important to Watson that he left the University of Copenhagen, against the terms of his fellowship, when he realized he needed the expertise of the Cambridge circle to make a real breakthrough. He did what was necessary and asked for permission later. What would’ve happened had he sat around waiting for permission?

The casual sexism with which Watson treats Rosalind Franklin, the expert in X-ray photography that wound up playing a major part in the discovery of the double helix, was striking to me reading the book in 2021. Watson tends to characterize her opinions and insights as obstinancy or rudeness. He doesn’t view his male colleagues the same way.

If cooperation is so critical to science, I can’t help but wonder what Watson could’ve achieved with a more collaborative attitude toward Franklin. Would the breakthrough have come even sooner? Would they have been able to make even more discoveries together if Watson had been more open to her expertise?

I loved getting a view of what is in a scientist’s mind as they make a major breakthrough. Watson was by no means certain he was right at first, but he worked methodically to prove what he suspected. That even such a genius has doubt in his ideas can cheer the rest of us!

The great chemist Linus Pauling had suggested a different structure of DNA, which turned out to be incorrect. But when he saw the elegance of Watson and Crick’s double helix, he was in awe and thrilled, rather than upset at being proven wrong.

I find that attitude to be one of the great things about science. There is both collaboration and competition, but in the end, everyone is working toward one goal: understanding.

In all, I found Watson’s book interesting and instructive. Since it was written in 1968, I’m not sure how many people are still reading it, but it’s worth a look. Check it out!

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I Just Went to a Lecture at the World’s Premier Genomics Institute. Here’s What I Learned.

This little guy has superpowers.

I just attended a fascinating talk from the Broad Institute at MIT on how the genomes of other species relate to our DNA as humans. Drs. Elinor Karlsson and Diane Genereux of the Broad Institute are intensively studying other mammals, working to uncover the genetic basis of their superpowers.

For example, the thirteen lined ground squirrel can hibernate, the teeth of the North American beaver include iron and are thus nearly indestructible, and the Jamaican fruit bat can eat all the sweets it wants without diabetes. What if we humans could do those things?

Any such applications are a long ways away, if ever, but learning about these incredible animals was definitely interesting.

Karlsson and her colleagues have sequenced the genomes of 240 species. Some spots on those genomes change little if at all, indicating they probably have a crucial function that can’t be changed. Others change faster than the normal rate of mutations, indicating a survival advantage to mutations in that area.

The researchers also noted that in the 100 million years since all mammals shared a common ancestor, every possible genetic mutation has been tried, given the base rate of random mutations. So, if we don’t see a mutation in living mammals, it probably was tried and failed. Dr. Karlsson likened this to nature’s clinical trial, an excellent analogy.

This genomic research is likely to have more and more applications because the cost of sequencing a human genome has dropped from $2.7 billion for the first one to under $1,000 now. The Broad Institute sequences one every 10 minutes.

This is an explosion of data similar to the development of the internet. It took years, but companies like Google came along and harnessed that data, with profound effects on society. I anticipate enormous advances will come from this research in the future.

If you want to watch the lecture in its entirety, it should be up on the Broad’s YouTube channel soon. And to register for future lectures like this, check out the Science for All Seasons website here.

Thanks to Drs. Karlsson and Genereux, moderator Tom Ulrich, and the Broad Institute for this awesome talk!

I Just Went to a Meeting With Two Experts on Gene Editing. Here’s What I Learned

Photo: Shengfang Jin, PhD, Vice President and Head of Discovery Biology, Editas Medicine

This morning, I attended an online presentation by two experts on gene editing and CRISPR. Shengfang Jin, VP of Editas Medicine, and Andrew Lin, who heads the Rare Disease Center at WuXi AppTec, presented a lot of great info, but what stuck out to me most was the trial Editas is doing to cure a rare eye disorder.

Leber congenital amaurosis type 10 generally causes people to be born blind or lose their sight by age 10. I can only imagine how difficult it must be. A new therapy from Editas targets this disease with a one-and-done treatment. This therapy is the first use of CRISPR in a human being, as opposed to a cell culture, and is currently in Phase 1/2 clinical trials.

Listening to Dr. Jin speak, I was put in mind of this passage from the Bible:

“the blind can see, the lame can walk, those who suffer from dreaded skin diseases are made clean, the deaf hear, the dead are brought back to life, and the Good News is preached to the poor. “

Matthew 11:5

I increasingly suspect we are at the dawn of a golden age in medicine. The rapid pace of COVID vaccine development or this first in vivo use of CRISPR may be the first signs of things to come.

P.S. The presentation should be up on the website of Genetic Engineering & Biotechnology News here soon.

What I Just Learned From a Discussion With Nobel Laureate Jennifer Doudna

Today I attended a fascinating discussion of the movie Human Nature, a documentary on CRISPR gene editing. Nobel Prize winner Jennifer Doudna, the co-inventor of CRISPR, gave us her perspective on the technology. Author Walter Isaacson and the filmmaker, Adam Bolt, also gave valuable insights.

Isaacson framed the moment well, saying that CRISPR is part of the 3rd great scientific revolution. The first was in the first half of the 20th century, in physics. The 2nd, in information technology, consumed the second half of the century. And in the 21st century, the revolution is and will be in the life sciences.

Because CRISPR can make DNA and RNA programmable like computer code, there’s a strong parallel between CRISPR and the IT revolution. What if biology and medicine progressed the way software has in the last few decades?

Doudna is particularly excited about the applications of CRISPR to cure cancer. CRISPR can be used to program the patient’s immune cells to attack tumors. This echoes what the co-founders of BioNTech said earlier this week at a call I attended.

Bolt and Doudna also noted that CRISPR was a scientific backwater at first. This really emphasizes the importance of funding basic science with no clear application. We never know where it will lead!

The first patient to be treated for sickle cell anemia using CRISPR is doing quite well a year later. Doudna and others are hard at work on further applications of this technology. As an investor and as a human being, I am eagerly anticipating these breakthroughs.

“Jennifer Doudna” by Koninklijke Nederlandse Akademie van Wetenschappen is licensed under CC BY 2.0

An Outstanding Science Movie and Your Chance to Talk to a Nobel Prize Winner

I just finished watching the superb documentary Human Nature, which details the origins and applications of CRISPR gene editing. The filmmakers interview all the leading people in the field and produce a fascinating and highly accessible narrative.

I found particularly striking how one of the co-inventors of CRISPR, Jennifer Doudna, enjoyed being in an unknown field at first but also wondered whether the field was neglected because it was a dead end. It shows us what we can accomplish when we overcome our self-doubt!

You can see the movie and attend a talk tomorrow at 1pm EST with Nobel Laureate Jennifer Doudna here, both free of charge. It’s not often a person gets the chance to ask questions of a Nobel prize winner. I’ll be there!

P.S. If you’re interested in CRISPR, I recommend this book.)

“Micah’s DNA” by micahb37 is licensed under CC BY-SA 2.0

How We Can Rewrite Our Genetic Blueprint

We may soon be able to edit our own genes using a new technology called CRISPR. It uses a bacteria to change data in the genetic code and could one day cure rare genetic diseases. There is also potential in editing genes to prevent cancer and heart disease.

In the new excellent book Editing Humanity by Kevin Davies, we learn how the technology was developed and how it has been applied and misapplied. CRISPR has cured a patient of sickle cell disease already, but was also used inappropriately to sloppily modify the genes of several babies in China. It’s tool with enormous potential, but that includes potential for abuse.

I am intrigued to see if this tool could one day be used to reverse the DNA errors that come with aging.

As an investor, it struck me that Beam Therapeutics, a company founded by David Liu and Feng Zhang, two of the best scientists in the field, went public at a valuation of only $180 million. If CRISPR doesn’t revolutionize health, perhaps it’s worth zero. But what if it succeeds? It could be the most valuable company in history. Indeed, since this book came out in mid-2020, I see the share price has increased nearly fivefold.

I also think we would benefit greatly from increasing our investment as a society in basic research. I was surprised to learn that the Howard Hughes Medical Institute supports the lab of one of the co-inventors of CRISPR, Jennifer Doudna, at a rate of $1 million per year. While HHMI’s work is outstanding and to be commended, I imagine Doudna could use a lot more than that. What would she do with $10 or $100 million a year? Our government and we as individuals (through crowdfunding) could get it to her and other leading scientists. The benefits we could reap can only be imagined.

Davies’ book is entertaining, readable, and informative throughout. I’d highly recommend it to anyone with an interest in CRISPR, gene editing, or health in general!