Unlocking Gene Therapy with Neil deGrasse Tyson & Gaurav Shah

How can innovative science lead to potential cures for people living with rare diseases across the globe? Neil deGrasse Tyson and comedian Chuck Nice learn about the science of gene therapy and its applications with Gaurav Shah, CEO, Rocket Pharma.

Learn how gene therapy works, tropism, and how rare diseases without a cure could potentially be history through advancements in science. We talk about how Gaurav’s interest in music helped his approach to science. Plus, we break down how they identify which genes and conditions to target and which viruses they use to deliver the new genetic material.

Why invest in seeking to cure rare diseases? Is having a rare disease itself rare? Learn about diseases they are targeting for gene therapy like Fanconi Anemia, LAD-I, Pyruvate Kinase Deficiency, and Danon Disease. Can gene therapy tackle chronic disease? How do scientists pick which genes to target?

What is the morality of gene editing in the future which could change the DNA of an entire individual? We discuss the implications and the ethics of different forms of gene therapy. Find out about the FDA’s role in approving this new medicine for the public and its risks.

Thanks to our Patrons Ernesto Rodriguez, James Lewandowski, Juan Cornejo, Shane, Hayden Christensen, jacob vine, and Calina Lungu for supporting us this week.

NOTE: StarTalk+ Patrons can listen to this entire episode commercial-free.

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Science meets pop culture on StarTalk! Astrophysicist & Hayden Planetarium director Neil deGrasse Tyson, his comic co-hosts, guest celebrities & scientists discuss astronomy, physics, and everything else about life in the universe. Keep Looking Up!

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00:00 - Introduction: Rocket Pharma
2:50 - The Intersection of Science & Art
6:28 - Gene Therapy 101
12:35 - Using Viruses for Medicine
16:34 - Treating Rare Diseases
21:44 - What Diseases is Gene Therapy Targeting?
27:06 - Ethics & Disability: What is Normal?
34:26 - Getting Gene Therapy Through The FDA
36:31 - Measuring Risk Factors

[StarTalk]

What was your favorite part of this episode?

[deucedaprodeuca]

I have an autoimmune disorder called cyclic neutropenia. My neutrophils disappear to zero once every 21 days and I get very sick and am sick for half of each month. Coincidentally, I started seeing an oncologist yesterday for the first time in many many years, and she got on the internet to try to find gene therapy, and today by coincidence, you're talking about it. There is no treatment for what I have, and all the gene therapy seems to be geared toward the more common disorders such as leukemia and sickle cell anemia. I think there are only 236 known cases for what I have.

[mervcharles]

“If you want everything to be “normal” that might eviscerate civilization of the most interesting people our genome could produce”

That statement by Neil is so profound. Our “limitations” drive our curiosity & motivate us to search for solutions.

[lisam]

The rare diseases used to be called orphan diseases because no one wanted to fund research into them. I'm glad to see that people are actually spending money on research to help. My sister died of an orphan disease (polychondritis) and there was very little known about it or treatments for it at the time. Here's hoping their research helps many, many people.

[anipush]

oh man! I'm on minute 3:40 and already getting chills

[Youknowmeozone]

Great episode .. interesting conversation

[SheSweetLikSugarNSavage]

Great matcha tea startalk time. 👍 It took 2 cups❤🍵 Enjoyed the public eavesdropping opportunity.

[production]

In Gaurav we trust..I bought Rocket stock..they are really onto something

[hervigdewilde]

Imagine the kind of experiments that companies could conduct on private orbital stations, beyond national boundaries and the reach of any laws. 😏
Interesting times ahead...

[Wilfoe]

I'm pausing just after the point where you discussed who decides what counts as normal to type this. I would argue that whether or not something is normal should be completely irrelevant to anything else. Regardless of who decides what is and isn't normal and what normal even means in any given day and age, it shouldn't matter. Rather than focusing on whether or not something is normal or accepted, we should be focusing on whether or not it is actually helpful, harmful, or neither. Not by merit of being the expected thing, but by merit of the actual method and results.

Literally every historical figure ever wasn't normal. Albert Einstein, Leonardo da Vinci, Gandhi. Them not being normal is a part of what got them into history! Literally every invention ever wasn't normal when it was first invented. The airplane, the train, the computer. People scoffed at those! Literally every form of progress that has ever occurred wasn't normal when it first occurred. Humanity learning how to harness fire, the discovery of electricity, the creation of entire countries. If people were so focused on being normal, there would be no progress! So why be normal?

Normal doesn't mean right or wrong or good or bad. Normal just means common, and there is nothing wrong with something being uncommon. There have been many points throughout history where normal has actually been bad. Slavery used to be normal in this country. It still is normal in a few other countries. Smoking used to be so normal that doctors claimed it had health benefits. Gun violence is currently so normal in this country that half of the nation views it as a fact of life. Normal is different from person to person, different from culture to culture, there is no set normal! Normal is effectively one of the least normal things someone could ever be. It is not normal to be normal. I'm not normal. That's not a bad thing.

Edit: Face blindness is called prosopagnosia. I have that one too. It's common with autism. An interesting benefit is that it's sometimes so severe that I can't even recognize what race someone is, so I'm less prone to racism.

[budzilladakilla]

Genius level intellect, wasting time watching instead of helping. I'm here.

[korstmahler]

Your buddy doesn't want to take the cure-pill because it worked out well for him. He's a scientist with a life.
Ask someone who's life has been ruined by their malfunctions instead. They'd take the pill in a heartbeat.

[commonsense]

And also your guess says that's up to the insurance companies I'm telling you I fight with the insurance companies and so do a lot of my friends often they refused to pay for even life-saving drugs.

I have a recent case with a family friend that the insurance health insurance company said they wouldn't pay for a life-saving drug they the person and the doctor appealed it and they were denied. Luckily they found a non-profit that would help cover the cost. Again the healthcare system in the United States is a crime that we don't make it right for all citizens to have health care they need and not have to worry about going bankrupt or dying are their children dying.😢 😢😢

[richardknapp]

I would approve of AI's future if it could clone Neil deGrasse Tyson.

[keigilmore]

I have Endometriosis and there is no cure! Spreading the word to find one for us humans

[GinaCarmichael-ndpt]

Yes however Dannon did not target COVID-19. The discovery updated clinical correlation due to cures for osteoarthritis, cancer, viruses, leukemia. Needs more human research. Trophism would require institutionalization allowing for the changes to be observed.

[cosmicmanik]

Read Dr. Stefan Lanka... it will blow your mind.

[Orisha]

This is how I am legend started... oh lawd. Ai or Zombies who you got? Who's gonna take us out?

[victorrutledge]

2.9 million Subscribers also care... never forget your audience. I personally want the virus which carries a payload that resets aging, to the end of the anabolic period of growth, and the beginning of the catabolic era. That way I get the best of both worlds.

[Anon-xdcf]

I have very few problems with gene thereapy...
I do however have a real problem with nanoparticle delivery systems.