Gene Therapy -- The time is now: Nick Leschly at TEDxBoston

"Forget the price. What's the value of that treatment?"
What if we could fix a patient's own cells and cure their disease once and for all instead of treating a chronic illness over their lifetime? The solution exists, but it's expensive. Nick Leschly, chief bluebird, explains how biotechnology can deliver corrective genes into a patient's diseased cells and disrupt the medical and insurance industries along the way

[@AngelusUmbra]

I spoke about gene therapy once to a relative and he used the usual words like, "unnatural," "playing God," and so on. I asked him, "if a doctor saves someone's life, do you believe he has played God by keeping one away from the afterlife? Couldn't advances in science and medicine actually be answers to prayers?"

[@exposingthecabal3115]

Ask them if they've ever taken or known anyone who has taken antibiotics, or had surgery. Both unnatural, both extend life. In the past those people would likely have died from an infectious disease, died without surgery, or at the least been left severely weakened or crippled without intervention.

[@666mikimiki]

having a simply healthy child...living a normal..healthy life...is not the same as ''designing super children''....plus we are nowhere close to advancments like that,simply cause its not a priority at the given moment, but it already is set into the future prospects, and we cant really change that. nothing new.

[@siramike2654]

those who thinks gene therapy is unnatural should first judge their mental capabilities. your God should be the one to stop doctors if he/she/it if ever exist. if you have capacity to leaf frog what nature have done for millions of years into just few days, it would be foolish of you not to take the opportunity. to hell with unethical or playing God. genetic diseases are random occurring things. one thing I do know is the life forms on earth including us are controlled by natural event. it is right for humans to reinvent themselves. Religious people should shut up and accept their stupidity.

[@siramike2654]

Miks, if one has capacity to create super human beings, then he/she has right to do. we should not leave lives to mercy of nature. remember what happened to dinosaurs could also happened to humans. dinosaurs did not choose to die from asteroid impact but due to lack of brains to deflect the asteroid coupled with bad luck sealed their fate.

[@mugensamurai]

Well said Thomas.

[@rmarbertin8131]

Actual production cost: $10-$20k "But can you put a price on your child's life? WE SURE CAN!!!"

[@prakash-nx8md]

The great responsibility of the young scientist is too make this treatment accessible to all sections of people VERY INFORMATIVE VIDEO SIR

[@MayurPanghaal]

yes sir...the society is ready for this...esp for those dying

[@ImiG01]

Whats cool is that this video was my homework!! And its a pretty decent video too. RIP Ethan :)

[@jakkuwolfinsomnia8058]

I produce this guy’s product. The challenges in making products is more challenging than it seems. Huge challenges that require skill, coordination, careful planning and a lot of money and support. But most importantly, it works and it’s fully possible and practical

[@JoeyR9669]

Sad that greed is the only thing hindering gene therapies progress at this point.

[@thelocustemperor]

5:10 - 11:40 The discussion really get's amazing!!

[@dr.hminga5043]

awsome presentation. moving. learnt a lot

[@liberty7188]

My. Child has a genetic disease. She has gene therapy and it has changed our lives. Its great for those who don't have hope. Please let's not judge but listen and think of those who loose their lives. The people who suffer and families who go through this.

[@rmarbertin8131]

Don't judge what, charging 10x the production cost? No, that's a crime against humanity.

[@alfonrizaldi5828]

This is Bot, don't believe this lie. Every vaccine is Poison.

[@darrenhines7810]

our health always comes down stakeholders involvement with money so that profit can be made for all again!

[@DrSharifulHalim]

Great lecture Nick!!

[@hsinsulete]

He is a showman,no substance,most of the work he presented were done by others, in particular doctors in Milan He comes across as very passionate but the PR he got with this TED presentation went to his head

[@alfonrizaldi5828]

So anyone dared to say mRNA couldn't affecting Genes??

[@bryanleggo3489]

This is not nRNA. Stop conflating the two.

[@ub2bn]

@@bryanleggo3489 True, but Transfer RNA (tRNA) simply serves as a link (or adaptor) between the Messenger RNA (mRNA) molecule and the growing chain of amino acids that make up a protein. Each time an amino acid is added to the chain, a specific tRNA pairs with its complementary sequence on the mRNA molecule, ensuring that the appropriate amino acid is inserted into the protein being synthesized. In both the case here, and with the mRNA Vax, the goal is to get the cell to produce Proteins, be they Spike Proteins or otherwise.

[@leahrsullivan]

Yes I'm ready for it.

[@athread9754]

What are the pros and cons of gene therapy?

[@nmccleary3889]

What an inspiring talk. Thank you for making me smarter

[@nathanterry45]

Thank you

[@michaelbarrett2968]

This is incredible

[@jakelopez3307]

Awesome stuff! Great video! Thank you for making such a strong contribution to the world!

[@darrenhines7810]

they make it sound so simple but forget all the other probable problems from putting foreign entities into the human body and the way the body will react thereafter in the short term and long term..he also said not all survive this gene therapy

[@Shuttergrrl]

Would love to see this therapy utilized for PKD/ARPKD. Keep doing amazing work! Also, it's not hemaglobin,...it's hemoglobin. It was spelled wrong on your slide presentation. Wish I could get a chance to be part of a biotech company like this. We are on the cusp of incredible treatment options.

[@adeelmehmood7439]

Me too diagnose pkd/adpkd diseases polycystic kidney disease. I hope if we will get luck to cure by biomedical

[@matthewjones7689]

Need to make one to fix mental illness

[@MayurPanghaal]

If we try to control this,many will find a way around the hurdles.China has already started human trials.Its a matter of time its widespread.

[@BiodegradeableMan]

Slowly but surely science is dealing with the nightmare hand God dealt us.

[@zannatul23]

can some1 please give me some good links that explain the gene therapy at a molecular level?? thank you!

[@needs2know1]

Interesting stuff. I'm actually thinking about getting into gene therapy. I think it will be the major treatment of the future.

[@Xilosphere]

If you go through with it, I strongly suggest looking into cosmetic gene therapy. Fewer people are born with rare genetic ailments than people discontent with aspects of their appearance. Namely hair texture, baldness, or skin/eye color. If you want a career that pays the bills, that's a future job waiting to be filled. Cheers.

[@gameplayplanet5015]

So do me a favor, find the cure for Stargardt's disease! That is the major cause of youth blindness and it is destroying me and my future =/

[@alaynawithluv]

Did you get into it?

[@bigglasses2625]

@@Xilosphere there are a lot of people with bad eyesight

[@rmarbertin8131]

I hope you invent a cure for greed.

[@AppleBottomNLP]

very informative

[@nicolareddwooddforest4481]

I am for stem cell research and gene therapy. However, those types of neurodegenerative diseases are new on planet Earth. Those illnesses are standing in direct correlation to the rise of environmental toxicity caused by pollution. Also eating meat, and in particular the meat of today which has been made toxic through hormone treatments, pollution, and vegan animals eating meat pellets in a perpetual cycle of rendering plant meat getting into the systems of vegan animals.

[@JackTekkel]

I'm an X-ALD patient. Cure me. I'm ready, you know where to find me, even willing to pay for it.

[@marinemomkerry]

+JackTekkel my son also, bone marrow 2008 are you on facebook ALD Support?

[@smartcatcollarproject5699]

This mostly opens the way for editing embryonic cells. For adults the problem would be about reaching cells inside the body. Of course everybody is speaking about blood stem cells, or sickle-cell disease, which are easily accessible to CRISPR/Cas-9 genome editing, as the targeted cells are in the blood. Just inject the solution, with some trans-membrane factor... but how do you access cells that are disseminated deep inside tissues, far from blood vessels ?

[@funny11744]

I have a genetic mutation on gene ADPRHL2 . Please specify the complementary DNA of mutation : ADPRHL2 gene : (NM_017825.2) c.[14C>T];[14C>T] (p.[Ala5Val];[Ala5Val]) Many thanks. It helps for CRISPR .

[@zerothehero123]

Viruses.

[@ayushupadhyay801]

I think we'll need new cell specific vectors

[@mandyd7064]

Amazing!!!!!!!!!!!!

[@jokedog]

8 years later....where are we with this?

[@SuperiorNo1]

Bureaucracy 😈

[@simonram8505]

Nowhere

[@biobagholder8081]

We have several gene therapies on the market. Bluebird has since gotten four drugs approved that are now on the market. The drug for ALD described here is expected to be approved in Europe in 2021 and in the US probably 2022-23.

[@shanemc8253]

...

[@maryanabardina6348]

I have recently started in the field of gene therapy. This video is really inspiring! Thank you for a great work and bringing hope to many patients.

[@liberty7188]

You are a hello. Please continue with the research.

[@422rutikanikam2]

This is so inspiring

[@sutopachakraborty6289]

Please help us,please. My daughter is attacked by AT.We're Bangladeshi.We're so helpless.

[@alexfrumusache7179]

This is a great video, and a very simple explanation for a very complex process. Thank you.

[@kamalkatial1530]

I am not human (as per him). Because I believe information should be absorbed without emotions.

[@9to5_]

And this was 9 years ago. I'm currently specializing in gene therapy.

[@zouli1000]

Where you study how I get involve?

[@samuelzev4076]

Will this treatment work for myopia?

[@ryanprotech9530]

The face you make when billions just agreed on this.

[@patrickgirondi1313]

This guy should be wearing a state uniform.

[@MayurPanghaal]

The end of traditional medicine as we know it.

[@bigglasses2625]

Good... ? I mean gene therapy is to cure genetic disease because traditional medicine can’t cure things that are genetic usually traditional medicine will still exist just not used for genetic disease to reduce pain.

[@dmeditz8100]

@@bigglasses2625 bro what if you are born cancer resistance to every carcinogen and also anti venom this new medicine era no more new generation vaccine if you want have babies in virus time plZz treat child with in-vitro gene therapy for corona virus.

[@konic40]

wait....did he just make the DNA chain longer? would the injected cell really proliferate like that...I thought the number you stay relatively constant, cells cant just divide whenever.

[@WgWilliams]

Yes, you put the corrected cells in the bone marrow and the bone marrow starts to copy and reproduce by self replicating the corrected cells.

[@nithinajames7628]

at least provide transcripts of the video. that would be really helpful

[@ludara8697]

So where are they now ?

[@mohsanzaki]

The ones who are suffering from genetic diseases should answer the questions, not the healthy ones who come up just about any excuse to be self righteous. Children have suffered enough from genetic disorders / diseases and its about time all of us should help put an end to it.

[@convictor11]

I have a rare genetic disease that hit me when i was 12.. it is not deadly but life altering, but sometimes i wish it was deadly i would probably get a chance at gene therapy ^^.

[@denaswords3571]

I have RP and ERM MY GENE IS PROM1 who can I talk to about a treatment ??

[@cuorenerazzurro1661]

8 years later and still no gene therapy on the market... Amazing.

[@SuperiorNo1]

Bureaucracy 😈

[@NDryuk]

There is some.

[@biobagholder8081]

Huh? Kymriah, Luxturna, Yescarta, Zolgensma...

[@dmeditz8100]

@@NDryuk human eithics only china has no eithics if in your experiment if a child is born disable who will be responsible for human life cost and also atleast a million of animals will be first test object.

[@jjhack3r]

Actually there is. It’s called the “covid 19 vaccine”

[@mahwishtahir5617]

I like this ted

[@jesusdailies3171]

Please I’ll be available if am to be the first person to use as a test for the method. Am haemopilia A

[@darrenhines7810]

barcode of life is our future according to these mad people!

[@user-sv4on7mo6t]

Gene therapy shows a very interesting twist in the modern ethical dilemma, and it does not seem to have a clear solution as to its remedy. I think this method of treatment is lacking in research funding and its potential is underestimated. We could potentially eliminate some genetic issues with simple mutations, especially deletion mutations, such as cystic fibrosis. There are many issues that face gene therapy, but it is possible with more research and time that we achieve effective therapy in humans or embryos. However, this video touches on yet another ethical dilemma. Will the cost of gene therapy be too high? What parent wouldn’t pay everything they had to save their child from a horrible life? Will only the children of the wealthy be allowed to cured from these diseases? Will only first world countries have this privilege? However, this argument could also have been used to stop the human genome project, but over time gene sequencing has improved to such a degree that it is cheap and available to everyone. Could this not also be the case for gene therapy?

[@pankajmohal189]

Can it cure genetic disorder causing MPPH Syndrome?

[@creativesolutions902]

Can this be done for retinitis pigmentosa? And other genetic diseases that cause blindness? I will Volunteer just let me know where and when… My son was just diagnosed and hes 14

[@biobagholder8081]

we are! For X-linked retinitis pigmentosa we have several gene therapy companies working through treatments! The most promising late stage one is probably Biogen's, which is about to finish their Phase 3 and likely enter the market in a year or two with good data. The most promising earlier stage ones are from AGTC and MeiraGTx.

[@ghostly9450]

eugenics

[@MayurPanghaal]

Dont try to stop this technology.Perfect it.Fast.Deliver it.Save lives.The planet can deal with human population.If we cant,there is always mars...or some other way.Go !!

[@Livio44]

should I study biochemistry or biotechnology to work in this field?

[@AllYourMemeAreBelongToUs]

Livio44 Either, lots of different medical professions are involved in gene therapy.

[@dracan69]

+Livio44 biochemistry and then you have to do a PhD and maybe 3 o 4 postdoc

[@rmarbertin8131]

The head researcher, University of Pennsylvania's Dr. Carl June, would probably be a good place to start.

[@rahulpaul2976]

Sir is there any kind of skin treatment available to change complexion genetically?

[@kloohooh]

why would he need chemo if he didn't have cancer?

[@rakeshdrreddys]

super

[@2663540]

Well lets see what the shareholders have to say about this.. Is this available to every social class? Think not.. So sorry to you all with minimum wages.

[@weimiao1272]

How do you prevent the immune attack even after the stem cell treatment?

[@weimiao1272]

***** I am not sure of your explain. There are 2 concerns, the cells and the artificial proteins. 1 Some GMO food can lead to allergy while the corresponding natural food do not cause any allergy. 2 The artificial proteins are allien to the host. If these proteins, not seen by the host before, trigger the immune system in the host, they will probably trigger the immune responses, because the therapeutic gene products are considered as intruders.

[@funny11744]

I have a genetic mutation on gene ADPRHL2 . Please specify the complementary DNA of mutation : ADPRHL2 gene : (NM_017825.2) c.[14C>T];[14C>T] (p.[Ala5Val];[Ala5Val]) Many thanks. It helps for CRISPR .

[@niranjanadhasarathan3913]

hi]

[@allorenzobancolita5029]

What is the difference of stem cell therapy and gene therapy?

[@indervirhundal7672]

Research and testing needed - Oh, stem cell treatment like companies like Giostar, advancells and Swiss Medico but gene therapy is important, too Could you not become a charity, which we can donate to? Offer for autism?

[@deatheffect575]

Great video. Cant help but to compare him to Sid off of Ice Age.

[@rmarbertin8131]

Because he's a weasel?

[@inspirediam]

did Ethan have chemotherapy?

[@1side1984]

What the fuck ever happened to doing medicine for the better of mankind! I swear it is always the smartest people who do the most horrible shit. Let one of their children ger sick; bet they get the treatment needed. We need to move away from this elitiest society bullshit.

[@aliciaridley17]

I didn’t watch it to voice and opinion I laughed at “I have 5 daughters, yes I’m looking for sympathy” 😆😆

[@g.a.m.e8126]

In the hands of Buck Rogers we're good, in the hands of Thanos...take a guess.

[@e61agem]

Does this guy remind you of Jerry Seinfeld?

[@Xilosphere]

If funding and ROI is such an issue, they should just work on cosmetic gene therapy to foot the bill. As an example, there are literally millions of African women (men?) that want manageable natural hair. Rest assured they'd be more than willing to pay thousands for a procedure that safely and permanently changes the shape of their hair follicles, so as to achieve a looser curl pattern. The same could also be said for those looking to change eye or skin color - traits of which I'd think are relatively easy to alter. It may seem an outlandish idea, but I think researchers can acquire a lot of the much needed capital with cosmetic manipulation. In doing so, we could at least exploit the inherent insecurities of humanity to not only make a better world for those with life-threatening genetic ailments, but to make people more comfortable in their own skin.

[@DS-rv2fc]

+Novakira Sato is it possible to change skin color by using gene therapy?

[@Xilosphere]

Strauss Field Yes, I would imagine it'd be relatively easy to alter pigmentation. It's just a matter of corporate support for an untapped market. I suspect the only thing getting in the way of these advancements are traditional views on ethics.

[@DS-rv2fc]

Novakira Sato I don't get it. People can change their hair color. Why would skin color change bother anyone?

[@AtheistEve]

+Daniel Narilton "The Sneetches" (and a number of other similar tales by Dr Seuss) explains in simple terms the problems with ego and socio-economics. Dr Seuss: essential reading.

[@rahulpaul2976]

Novakira Sato is there any kind of skin treatment available to change complexion genetically?

[@ejrev]

"and then start Proddatur cells all those daughter cells" 10:17. what does Proddatur mean?

[@GeneralLee131]

He said something like "prod daughter cells". I think by "prod" he was truncating "produce".

[@felixromano3091]

They should use this to treat hair loss or Ed it would be approved the next day cuz of all the old dudes in government

[@immigrantworld4899]

Gene therapy for red green color blindness please.., I have been waiting for human trials since 2009

[@adanetilahune9645]

the current research going to treat genetic disease now ,if you develope biotechnology it is better one to treat genetic disease

[@iamkenntron]

It doesn't pay very well

[@jackysub1941]

I have a question, and I would appreciate anyone with relevant knowledge to answer. So how does gene addition or gene editing actually work? Wouldn't our body recognize the changes to the DNA as a 'mutation' and try to fix the change (since there are still other copies of the gene in 23 pairs of chromosomes). And even if we got it to work, how do we apply the treatment to every individual cells in our body? I am really confused. Plz some one help me!

[@chrism45]

It's been over a year but someone else who ends up on this comment might benefit from an answer. In the video they showed an integrating virus (modified from HIV) so it is just added permanently to your own DNA (DNA is DNA there is no yours or mine it's a chemical). To answer your question, how is it not recognised as foreign? Well immune response cells recognize other cells by their surface. The surface in this case was the patient's own stem cells that were previously harvested. At the DNA level, for example when a virus enters the cell and releases its RNA or DNA the cell has some defense mechanisms in the form of enzymes to degrade them called DNases and RNases. Unfortunately, there are no DNases and RNases in the nucleus so once it reaches and enters the nucleus it can use DNA polymerase to multiply therefore more of it will be made, while it is continually destroyed outside the nucleus by DNases and RNases. For your question about targetting specific cells instead of every one, we usually add a tissue-specific promoter. This means for example this new gene will only be active in the heart and nowhere else. However, sometimes it doesn't matter and you just express it everywhere and it works fine just like in the very recent Zolgensma drug for SMA. These are some of the reasons gene therapy research is slow: delivery, efficiency and keeping toxicity low. Much like the video said, some of the cells were made into macrophages and only a proportion of that managed to enter the blood-brain-barrier. In practice it's much harder than it sounds to just put the gene, it usually never reaches its destination and is just destroyed by the liver, never enters the cell, if it does it's usually degraded and if it's not it's usually not enough to show significant effect on the patient.

[@shanemc8253]

@@chrism45 crazy with theses covid gene therapys

[@toxicgamesorg]

as any one heard of ,the venus project.

[@localbrownboy]

I have Thallasemia, this treatment MUST advance.

[@DJ-Illuminate]

Someone way more advanced than us must have designed everything. Even trying to copy DNA for programming makes programming advance by 100 years. Reminds me of reverse engineering alien technology but instead of a Roswell crash we are all walking around in our own personal UFO.

[@jasmijnluyten3252]

6tw gang

[@AxelSituation]

Why does everyone still think its okay to make jokes about how awful it is to have daughters??? Then don't have children! And as a PhD biochemist, this presentation is not wholly accurate, outdated, and nothing new. Apparently anyone can give a ted talk these days.

[@Bleeinyourself]

I'm a pharmaceutical company and I disapprove.

[@bionh]

disagree. humor is necessary to keep it from being too sad

[@inspirediam]

Is the pharmacutical business willing to allow a treatment that will cure, thus the need for thier bandaid solutions will no longer be needed, and their business will cease to exist.

[@Afflictamine]

not so 'cool' now is it ?

[@nickirhododendron7569]

Yes to gene therapy. No to animal testing. So test on humans instead. Dr. Jackyll and Mr. Hide.

[@naikchiya7107]

seller.indiamart.com/product/manageproduct/

[@ShrimpTechStocks]

Voice = Gavin Newsom

[@yourchannelname325]

"The time is now" - 10 years later - No it isn't