Medicine's Big Breakthrough: Editing Your Genes

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0:00:04 > 0:00:08Tonight on Panorama, the scientific breakthrough that could

0:00:08 > 0:00:11change the lives of everyone and everything on the planet.

0:00:13 > 0:00:18It's an advance in gene editing, which holds out the promise of cures

0:00:18 > 0:00:22and personalised treatments for some of our most deadly diseases.

0:00:23 > 0:00:27Imagine a tool that allows scientists to change

0:00:27 > 0:00:30the letter code in the DNA of a cell

0:00:30 > 0:00:34so precisely that we could change a single base pair in

0:00:34 > 0:00:37the 3 billion base pairs of the human cell.

0:00:37 > 0:00:42It sounds complex but gene editing has just been made simple

0:00:42 > 0:00:46and is revolutionising research into life's big killers

0:00:46 > 0:00:48and the diseases of ageing.

0:00:49 > 0:00:51Gene editing has created a fundamentally new

0:00:51 > 0:00:53kind of medicine,

0:00:53 > 0:00:56and this means that we can now treat genetic disease,

0:00:56 > 0:00:59infectious disease and cancer in ways that, ten years ago,

0:00:59 > 0:01:01would have seemed like science fiction.

0:01:01 > 0:01:04It crosses the animal and plant kingdoms

0:01:04 > 0:01:08and kick-starts a new era of genetically modified organisms.

0:01:10 > 0:01:12We can now control evolution

0:01:12 > 0:01:17so precisely that insects which spread disease could be eradicated.

0:01:21 > 0:01:24But medicine's big breakthrough is not without risk.

0:01:38 > 0:01:42I've set out to discover how gene editing could change our world.

0:01:44 > 0:01:45It's just four years

0:01:45 > 0:01:49since researchers discovered a new technique to edit DNA called

0:01:49 > 0:01:53CRISPR, which is so fast, cheap and accurate,

0:01:53 > 0:01:57it swept through nearly every field of scientific research.

0:01:59 > 0:02:03I've come to the West Coast of the United States to San Francisco

0:02:03 > 0:02:08to meet a pioneer in this fast-moving field of science -

0:02:08 > 0:02:10one of the co-discoverers of CRISPR.

0:02:15 > 0:02:17The University of California, Berkeley.

0:02:19 > 0:02:24The pace of science has just increased incredibly.

0:02:24 > 0:02:26'Jennifer Doudna is a biochemist

0:02:26 > 0:02:31'and now one of the world's most influential scientist.

0:02:31 > 0:02:35'She's tipped to share a Nobel Prize for discovering

0:02:35 > 0:02:37'a new form of gene editing.'

0:02:38 > 0:02:42CRISPR's an acronym and it stands for clusters of regularly

0:02:42 > 0:02:46interspaced short palindromic repeats. Big mouthful!

0:02:46 > 0:02:50- Easier to say CRISPR.- Don't let the terminology put you off.

0:02:50 > 0:02:55Put simply, the CRISPR system acts as a chemical cleaver which

0:02:55 > 0:03:00allows scientists to alter any form of life.

0:03:00 > 0:03:02This is the thing that's so exciting.

0:03:02 > 0:03:05Laboratories around the world have adopted this

0:03:05 > 0:03:10technology for applications in animals, plants, humans,

0:03:10 > 0:03:13fungi, other bacteria - essentially,

0:03:13 > 0:03:16any kind of organism that labs are studying.

0:03:18 > 0:03:22In Boston, a world-leading geneticist believes CRISPR

0:03:22 > 0:03:25heralds a breakthrough for transplant patients,

0:03:25 > 0:03:29growing their organs in animals - a revolution in science.

0:03:31 > 0:03:34The power that we have now is almost limitless.

0:03:34 > 0:03:38CRISPR is one of the few technologies that works first time.

0:03:38 > 0:03:42There's almost no field of medicine, agriculture

0:03:42 > 0:03:46and ecosystems that will be unaffected.

0:03:50 > 0:03:56So what is gene editing? This is the Francis Crick Institute in London.

0:03:56 > 0:04:00When it opens in a few months, it will be the biggest

0:04:00 > 0:04:05biomedical laboratory in Europe and will be a centre for gene editing.

0:04:07 > 0:04:10Inside each cell in our body is our genome -

0:04:10 > 0:04:13billions of pieces of genetic code.

0:04:13 > 0:04:18It's the blueprint or instruction manual for life.

0:04:18 > 0:04:23A single error or spelling mistake in that DNA can trigger disease.

0:04:23 > 0:04:26There are thousands of genetic disorders

0:04:26 > 0:04:30and many more conditions that develop as we age.

0:04:30 > 0:04:35CRISPR gene editing enables scientists to scan the entire genome

0:04:35 > 0:04:38and then, using molecular scissors,

0:04:38 > 0:04:44to cut both strands of DNA and delete, insert or repair the code.

0:04:49 > 0:04:53One of the first targets is type I diabetes -

0:04:53 > 0:04:56a condition that affects ten-year-old Jack,

0:04:56 > 0:04:59who's from Minnesota in the Midwest United States.

0:04:59 > 0:05:03His pancreas doesn't produce the hormone insulin, which controls

0:05:03 > 0:05:09blood sugar levels, so his dad, Chris, has to keep a careful watch.

0:05:09 > 0:05:11This is one of the challenges that you have

0:05:11 > 0:05:14when you take care of a type I diabetic, is that you end up

0:05:14 > 0:05:20checking blood glucose 10-12 times a day to manage healthy blood sugars.

0:05:20 > 0:05:24So we take turns at night, checking at either 2.30 or 5.30,

0:05:24 > 0:05:27to make sure that his blood glucose doesn't go low at night.

0:05:27 > 0:05:32So you or your wife has to get up at 2.30 or 5.30 every morning,

0:05:32 > 0:05:36- 365 days a year? - That's exactly right, yeah.

0:05:36 > 0:05:39- And Jack never wakes up? - No, not at all.

0:05:39 > 0:05:41What can happen to you if you don't take action?

0:05:41 > 0:05:45Um, well, first of all, I could faint,

0:05:45 > 0:05:50and then, that's why I bring a Buddy with me when I'm at school

0:05:50 > 0:05:54and I feel low, so I don't faint and then no-one knows.

0:05:58 > 0:06:03Chris Burlak hopes gene editing might cure his son.

0:06:03 > 0:06:06He's an immunologist at the University of Minnesota.

0:06:08 > 0:06:09Come on, Jack.

0:06:11 > 0:06:13Nice job, Jack.

0:06:18 > 0:06:22In type I diabetes, insulin-producing cells

0:06:22 > 0:06:26in the pancreas called islets gradually die.

0:06:26 > 0:06:30Islet transplants are possible but limited

0:06:30 > 0:06:33because of a worldwide shortage of donor organs.

0:06:34 > 0:06:38Chris and his team believe the answer could come from pigs.

0:06:38 > 0:06:41We sequence genes, we sequence our PCR reactions

0:06:41 > 0:06:43and we construct the CRISPR...

0:06:43 > 0:06:47'They're aiming to delete some key genetic markers that identify

0:06:47 > 0:06:49'the pig cells as foreign

0:06:49 > 0:06:53'so that the human immune system won't reject the transplant.'

0:06:54 > 0:06:59So what we're looking at here are pig cells that we've cloned

0:06:59 > 0:07:02and then gene edited using the CRISPR technology.

0:07:02 > 0:07:06- So you're trying to make them more humanlike?- That's correct.

0:07:06 > 0:07:09So being like stealth islets means that they won't get

0:07:09 > 0:07:12recognised by the human immune system during transplantation.

0:07:12 > 0:07:14And for some people with type I diabetes,

0:07:14 > 0:07:16could this potentially be a life-saver?

0:07:16 > 0:07:18This could be a life-saver, for sure.

0:07:18 > 0:07:20People who have suffered from type I diabetes

0:07:20 > 0:07:25who have secondary complications that damage their nerves,

0:07:25 > 0:07:30impair their vision, cause kidney failure or cardiovascular disease.

0:07:31 > 0:07:37Human trials are some way off, but if it works, then patients like Jack

0:07:37 > 0:07:39could have a tissue-matched treatment

0:07:39 > 0:07:41from gene-edited pig cells.

0:07:43 > 0:07:48And, Jack, how cool would it be if it was your dad that found a cure?

0:07:48 > 0:07:52It would be so amazing.

0:07:52 > 0:07:57It'd be 5 million times amazing...because he's my dad.

0:07:57 > 0:07:58Aw, that's sweet.

0:08:06 > 0:08:10Gene editing has revitalised the whole concept of

0:08:10 > 0:08:13cross-species transplantation.

0:08:13 > 0:08:16Although pig organs are of a similar size to ours,

0:08:16 > 0:08:20the human immune system would instantly reject them

0:08:20 > 0:08:23and there have been fears that such transplants could allow

0:08:23 > 0:08:27diseases to jump across the species barrier.

0:08:27 > 0:08:31Now gene editing offers the hope of solving both problems.

0:08:31 > 0:08:35Scientists envisage organ farms of the future, providing

0:08:35 > 0:08:40an endless supply of hearts, lungs, livers and kidneys for transplant.

0:08:46 > 0:08:50What you are seeing here is the mixing of two species -

0:08:50 > 0:08:52pig and human.

0:08:52 > 0:08:56This pig embryo is being injected with human stem cells

0:08:56 > 0:08:59by a team at UC Davis in California.

0:09:02 > 0:09:05- So these are the human cells...- Yes.

0:09:05 > 0:09:09- ..going down the tube into the pig embryo?- Exactly.

0:09:09 > 0:09:15The idea is that these cells will integrate into this embryo,

0:09:15 > 0:09:19and then we'll transfer this embryo to a recipient,

0:09:19 > 0:09:23to a female, and allow it to develop past this stage.

0:09:26 > 0:09:29The pig embryo was gene edited using CRISPR to delete

0:09:29 > 0:09:34the DNA instructions to create a pancreas.

0:09:34 > 0:09:37The ambition is the human cells will fill the void

0:09:37 > 0:09:40and grow a human pancreas inside the pig.

0:09:43 > 0:09:47Our hope is that this pig embryo will develop normally

0:09:47 > 0:09:52but the pancreas will be made up almost exclusively

0:09:52 > 0:09:55out of human cells, so that then that pancreas could be

0:09:55 > 0:09:59compatible with the patient for transplantation.

0:09:59 > 0:10:00PIGS GRUNT

0:10:00 > 0:10:03This is the farm where we keep the animals after we've done

0:10:03 > 0:10:04the embryo transfer.

0:10:04 > 0:10:07'Just like the earlier diabetes research we saw,

0:10:07 > 0:10:10'this is an attempt to produce pancreatic tissue

0:10:10 > 0:10:14'in pigs that the human immune system won't reject.'

0:10:16 > 0:10:21The embryos carried by these sows are known as chimeras.

0:10:21 > 0:10:22In Greek mythology,

0:10:22 > 0:10:27Chimeras were monsters made from a mixture of animals.

0:10:27 > 0:10:31Regulators are concerned where the human cells might end up

0:10:31 > 0:10:35in the embryo, perhaps even altering the pig brain.

0:10:36 > 0:10:38We want to prevent that.

0:10:38 > 0:10:42We think that that potential is very low, in part because of

0:10:42 > 0:10:47the whole architecture, size and composition of the pig brain.

0:10:47 > 0:10:50We don't expect a human brain growing

0:10:50 > 0:10:55but that's something that we want to support with scientific information.

0:10:56 > 0:11:00This research raises profound ethical concerns - crucially,

0:11:00 > 0:11:06just how human are the piglets developing inside these sows?

0:11:06 > 0:11:10It's such a sensitive area that the chimeric embryos will not be

0:11:10 > 0:11:15allowed to go to term but be removed after 28 days' gestation

0:11:15 > 0:11:20for tissue analysis when they're still about half an inch long.

0:11:26 > 0:11:30And a team in Boston has addressed another huge obstacle to

0:11:30 > 0:11:36cross-species transplants - that pig diseases might infect humans.

0:11:36 > 0:11:41They used CRISPR to delete dozens of copies of an animal virus

0:11:41 > 0:11:43embedded in the pig's DNA.

0:11:44 > 0:11:48It opens up the possibility of not just transplantation from pigs

0:11:48 > 0:11:54to humans, but the whole idea that a pig organ is perfectible.

0:11:54 > 0:11:59Do you envisage that we will have pig organ farms that will

0:11:59 > 0:12:04yield unlimitless supply of tissue for human transplantation?

0:12:04 > 0:12:05Absolutely.

0:12:05 > 0:12:10We have a huge shortage now which is getting worse,

0:12:10 > 0:12:13and so this would be very clean and on demand

0:12:13 > 0:12:16so that they're very healthy when the surgeon gets them.

0:12:21 > 0:12:24But this is also a story in the here and now.

0:12:24 > 0:12:27Patient trials are already underway involving

0:12:27 > 0:12:29an older form of gene editing.

0:12:29 > 0:12:34Scientists are focusing on blood and immune disorders because

0:12:34 > 0:12:39faulty cells can be removed from the body, corrected and then put back.

0:12:39 > 0:12:44It provides a proof of principle that gene editing can treat disease.

0:12:55 > 0:13:00San Francisco - a centre of gay culture in the United States.

0:13:04 > 0:13:09In the early '80s, it was one of the first places to identify AIDS.

0:13:13 > 0:13:17The Castro District was particularly badly hit.

0:13:17 > 0:13:21Thousands of mostly gay men were infected with HIV -

0:13:21 > 0:13:24a virus for which there was no treatment.

0:13:27 > 0:13:32Ever since, it's been a focal point for the fight against HIV AIDS.

0:13:34 > 0:13:40It was a holocaust of young, delightful, gay men dying

0:13:40 > 0:13:42miserable, painful deaths of AIDS.

0:13:44 > 0:13:46We had 2-3 patients dying a week in this office

0:13:46 > 0:13:51and it's been a sea change where, 27 years later,

0:13:51 > 0:13:56HIV is basically a stable, chronic, manageable illness.

0:13:56 > 0:14:01Jacob Lalezari is a veteran of the fight against AIDS.

0:14:01 > 0:14:04He's been running clinical trials here at the Quest Clinic

0:14:04 > 0:14:06since the late '80s.

0:14:06 > 0:14:07KNOCKING

0:14:08 > 0:14:12- Hey there.- Hey, how you doing? - I'm good, yourself?- Good to see you.

0:14:12 > 0:14:14Um, so I was just looking at your chart,

0:14:14 > 0:14:17and actually, I like what I'm seeing.

0:14:17 > 0:14:19Your T-cell count's still about 500.

0:14:20 > 0:14:24Matt is one of around 80 HIV patients who've

0:14:24 > 0:14:27been on the world's first gene editing trials.

0:14:28 > 0:14:32This personalised treatment involved taking immune cells

0:14:32 > 0:14:33from their blood.

0:14:34 > 0:14:39Doctors deleted a gene to replicate a rare genetic trait

0:14:39 > 0:14:44carried by a few people which makes them resistant to HIV infection.

0:14:46 > 0:14:49- So, Matt, how did that go? - It was really interesting, you know.

0:14:49 > 0:14:55My lab values look really good, my viral load is pretty good,

0:14:55 > 0:14:56pretty well controlled.

0:14:56 > 0:14:59I mean, that's kind of the point of the study - to see

0:14:59 > 0:15:05how well you can naturally control HIV after you get the treatment.

0:15:05 > 0:15:08And how long have you been off your meds?

0:15:08 > 0:15:11- I've been off my meds for two years. - That's pretty amazing.

0:15:11 > 0:15:13It is pretty amazing.

0:15:15 > 0:15:19You have been at the forefront of HIV trials now.

0:15:19 > 0:15:23Since then, how, in general terms, have those studies gone?

0:15:23 > 0:15:24It's too early to say for sure

0:15:24 > 0:15:29whether gene therapy is going to be the key component of HIV cure

0:15:29 > 0:15:33or whether maybe it might be a component in combination with

0:15:33 > 0:15:37other therapies that specifically address the viral reservoir.

0:15:37 > 0:15:41And what would it mean for you if we got to a cure?

0:15:41 > 0:15:45I'm planning my retirement around an HIV cure.

0:15:45 > 0:15:51After 27 years, I've had a bellyful and I can't wait to hang it up.

0:16:02 > 0:16:04Cafe Flore in the Castro District.

0:16:04 > 0:16:07Matt's been coming here since the late '80s

0:16:07 > 0:16:12and met up with two fellow survivors from the AIDS epidemic.

0:16:12 > 0:16:15You know, when I sit here, I can name ten,

0:16:15 > 0:16:1815, 20 people that I used to talk with,

0:16:18 > 0:16:22the very thing we're talking about now - how do we get to a cure -

0:16:22 > 0:16:24that are all dead.

0:16:24 > 0:16:28We're sitting in a restaurant where people used to come

0:16:28 > 0:16:31and dump ashes of their loved ones.

0:16:31 > 0:16:34So we're sitting in, essentially, a graveyard.

0:16:34 > 0:16:37You guys have known each other over 20 years.

0:16:37 > 0:16:42What is it like to be very much at the heart of the search for a cure?

0:16:42 > 0:16:45It's definitely personal being here in the city,

0:16:45 > 0:16:48in the centre of where a lot of the research is happening,

0:16:48 > 0:16:54where a lot of friends and family and lovers have passed away.

0:16:54 > 0:16:58I have a friend who's 23, recently infected with HIV,

0:16:58 > 0:17:01and when I talk to him, I tell him, you know,

0:17:01 > 0:17:06take your medications, take care of yourself, because you will be cured.

0:17:10 > 0:17:14The next gene editing trial will be in patients with the serious

0:17:14 > 0:17:17blood clotting disorder haemophilia.

0:17:17 > 0:17:20The treatments were designed here by the biotech firm Sangamo,

0:17:20 > 0:17:22which also did the HIV studies.

0:17:24 > 0:17:27The joy of editing is DNA becomes a drug target.

0:17:29 > 0:17:33We can approach the human genome and change it, essentially, at will.

0:17:34 > 0:17:37There's good hope that in your and my lifetime,

0:17:37 > 0:17:41genetic diseases of the bloodstream will be very significantly

0:17:41 > 0:17:45diminished, that we will have, essentially, cured.

0:17:46 > 0:17:49Many people carry genetic traits that make them

0:17:49 > 0:17:52less susceptible to certain diseases.

0:17:52 > 0:17:56It might be possible to develop these into a genetic vaccine

0:17:56 > 0:17:58so that everyone could benefit.

0:18:00 > 0:18:05We know what the disease protective signatures are in human DNA

0:18:05 > 0:18:08that cause people to be resistant to cardiovascular disease

0:18:08 > 0:18:10and neurodegenerative disease.

0:18:10 > 0:18:15And as technology develops, I see no fundamental obstacle to,

0:18:15 > 0:18:17if you wish, a vaccine.

0:18:17 > 0:18:20I think gene editing as a vaccination against

0:18:20 > 0:18:23cardiovascular or neurodegenerative disease is not futuristic.

0:18:28 > 0:18:31Gene editing also raises the extraordinary

0:18:31 > 0:18:36possibility of eradicating diseases like malaria, dengue fever

0:18:36 > 0:18:41and the Zika virus, by targeting the mosquitoes which carry them

0:18:41 > 0:18:44with what are known as gene drives.

0:18:47 > 0:18:50Scientists do this by inserting an artificial gene

0:18:50 > 0:18:54into the DNA of mosquito embryos that will make

0:18:54 > 0:18:58an increasing proportion of female offspring sterile.

0:18:58 > 0:19:03The gene drive is embedded in the DNA to ensure the changes

0:19:03 > 0:19:07are inherited, unlike natural evolution, where chance is involved.

0:19:07 > 0:19:09Within a few years,

0:19:09 > 0:19:12an entire species of mosquito could be eradicated.

0:19:17 > 0:19:20The research is not taking place in Africa but in London,

0:19:20 > 0:19:25in a sealed basement laboratory at Imperial College,

0:19:25 > 0:19:26just yards from the Science Museum.

0:19:30 > 0:19:34Rather than using insecticides, which kill multiple organisms,

0:19:34 > 0:19:39gene drives could pursue any one of the thousands of species

0:19:39 > 0:19:43of mosquito down the generations to extinction.

0:19:44 > 0:19:46This can be a very powerful technology

0:19:46 > 0:19:48because the mosquitoes do the work.

0:19:48 > 0:19:51We're making sure that we build the gene drive to

0:19:51 > 0:19:54work as efficiently as we possibly can.

0:19:54 > 0:19:59But a genetic destruct button would raise concerns about possible

0:19:59 > 0:20:03unintended consequences on the ecosystem.

0:20:03 > 0:20:05People are right to have concerns with any new technology.

0:20:05 > 0:20:10We need to make sure that there are no ecosystem consequences.

0:20:10 > 0:20:13Given that there are hundreds of thousands of people,

0:20:13 > 0:20:18mostly children, in Africa who die from malaria infection every year,

0:20:18 > 0:20:23why not go ahead and introduce this and see if it can save lives now?

0:20:23 > 0:20:26Well, I mean, the temptation is to use it as soon as you've got it,

0:20:26 > 0:20:30but I think it would not be ethical to throw something in there that's

0:20:30 > 0:20:33not tested as much as you possibly can, which is what we're doing here.

0:20:43 > 0:20:47Gene editing is already allowing scientists to take ownership

0:20:47 > 0:20:52of natural selection and to make radical changes to farm animals.

0:20:55 > 0:20:58Let me show you a powerful example of gene editing.

0:20:58 > 0:21:02Many breeds of cattle have horns, like these Herefords here,

0:21:02 > 0:21:07but scientists took the genetic variation for hornless cattle from

0:21:07 > 0:21:12the Black Angus you could see there to produce these - hornless cattle.

0:21:15 > 0:21:18Currently, millions of dairy cattle have their horns

0:21:18 > 0:21:20physically removed each year.

0:21:22 > 0:21:25It's a fairly painful procedure where typically

0:21:25 > 0:21:28the horn buds are treated with lidocaine and then burnt off.

0:21:28 > 0:21:32And it's not pleasant for the animals or the farmers.

0:21:32 > 0:21:36The advantages are you can basically go into a single animal and make

0:21:36 > 0:21:40a number of changes in genes that you know are superior,

0:21:40 > 0:21:43rather than having to cross it in from different animals.

0:21:43 > 0:21:46So it basically accelerates the rate that you can make

0:21:46 > 0:21:48genetic improvement.

0:21:48 > 0:21:52But animal welfare groups warn gene editing could create unforeseen

0:21:52 > 0:21:57problems and is only needed where you have intensive farming.

0:21:57 > 0:22:00I look at the benefits of this technology, and to me,

0:22:00 > 0:22:04that outweighs any potential risks, which in this case I think

0:22:04 > 0:22:06are very minimal cos we've actually

0:22:06 > 0:22:09brought in the variant from a different cow breed.

0:22:09 > 0:22:13And so we've been eating that variant for hundreds of years.

0:22:13 > 0:22:17So I don't see food-safety risks, I just see an animal-welfare benefit.

0:22:23 > 0:22:27And you don't need a science degree to do gene editing.

0:22:29 > 0:22:34Manipulating DNA is now so simple that many people are trying

0:22:34 > 0:22:38DIY gene editing at home and buying their kits from here.

0:22:38 > 0:22:39HE KNOCKS

0:22:42 > 0:22:45- Hi, Josiah.- Hey, Fergus. - Good to see you.- Nice to meet you.

0:22:45 > 0:22:50- So this is your CRISPR gene editing lab?- Welcome to my lab.

0:22:50 > 0:22:53How difficult is it? Would you be able to show me how to do it?

0:22:53 > 0:22:55Yes, I could definitely show you how to do it.

0:22:55 > 0:22:59It's something you could learn almost as simply as driving a car.

0:22:59 > 0:23:01Everything somebody would need.

0:23:01 > 0:23:06'He showed me the CRISPR kits he sells, starting at around 140.'

0:23:06 > 0:23:10We have DNA that you'll need to do the experiment.

0:23:10 > 0:23:11There's a lot of things people can do.

0:23:11 > 0:23:15We like to promote a lot of things related to food and brewing.

0:23:15 > 0:23:17A lot of people use yeast to bake,

0:23:17 > 0:23:20to brew - completely safe and

0:23:20 > 0:23:23it's actually a genetic engineering tool that a lot of scientists use.

0:23:23 > 0:23:28Or engineer a yoghurt, right, to have different flavours.

0:23:28 > 0:23:31It feels like I'm in an episode of MasterChef!

0:23:31 > 0:23:32THEY LAUGH

0:23:32 > 0:23:35- All right. Are you ready to do an experiment?- OK, I'm ready.

0:23:35 > 0:23:39- All right. So first, you need to put on one of these.- OK.

0:23:40 > 0:23:43- Why do I need to wear that? - Just to make you look silly!

0:23:43 > 0:23:44THEY LAUGH

0:23:47 > 0:23:50So these are tubes which we're going to put

0:23:50 > 0:23:54- the chemicals that we need to do the experiment.- Yep.

0:23:54 > 0:23:57Now, you can see the bacteria on there. They're white.

0:23:57 > 0:24:01Just move your loop over it and fill up with some bacteria.

0:24:02 > 0:24:07- And now we're going to break up any of the clumps. Looks good.- Yeah.

0:24:07 > 0:24:11Now we're ready to add some DNA.

0:24:11 > 0:24:14We need this tube, which has part of the CRISPR system.

0:24:14 > 0:24:17And once they get the DNA inside their cells,

0:24:17 > 0:24:20then the whole genetic engineering process will take place.

0:24:20 > 0:24:23So this really is democratising science.

0:24:23 > 0:24:26Yes, it's really that simple.

0:24:26 > 0:24:30So this media normally wouldn't let the bacteria grow,

0:24:30 > 0:24:33but now, after editing its genome, you can

0:24:33 > 0:24:37see the little bacterial colonies, the white dots on there.

0:24:37 > 0:24:38Yeah, wow.

0:24:38 > 0:24:4330 years ago, people were taking computers, starting companies

0:24:43 > 0:24:46with computers, and just completely changing the world.

0:24:46 > 0:24:48And the fact that this is going on with synthetic biology

0:24:48 > 0:24:52and genetic engineering is amazing, right?

0:24:52 > 0:24:57The next amazing company could come out of a two-car garage

0:24:57 > 0:25:00in the San Francisco Bay area or anywhere, anywhere in the world.

0:25:05 > 0:25:09But it is gene editing in human embryos which raises

0:25:09 > 0:25:11the biggest ethical concerns.

0:25:11 > 0:25:17This might cure inherited disease or add in genetic enhancements,

0:25:17 > 0:25:20paving the way for designer babies.

0:25:20 > 0:25:24A team here, at the Francis Crick Institute in London, has been

0:25:24 > 0:25:29given permission to do gene editing in one-day-old human embryos,

0:25:29 > 0:25:31but purely for medical research.

0:25:34 > 0:25:35Kathy Niakan,

0:25:35 > 0:25:40named by Time magazine as one of the world's 100 most influential

0:25:40 > 0:25:45people, will use CRISPR to edit out key genes from the embryo to

0:25:45 > 0:25:50try to identify the genetic faults which lead many women to

0:25:50 > 0:25:51repeatedly miscarry.

0:25:52 > 0:25:54What I'm hoping is that it provides us

0:25:54 > 0:25:58with really crucial insights into early human development.

0:25:58 > 0:26:03The UK is the first country to formally approve gene editing in

0:26:03 > 0:26:08human embryos, which will be allowed to develop for just a few days.

0:26:09 > 0:26:13I think it could help in identifying ways in which

0:26:13 > 0:26:19we could improve IVF, to identify those embryos that are likely

0:26:19 > 0:26:24to continue to develop and thrive and give rise to healthy babies.

0:26:24 > 0:26:27And in terms of miscarriage, it could help us

0:26:27 > 0:26:31to identify some of the underlying molecular basis of why

0:26:31 > 0:26:35certain embryos do not go on to develop successfully.

0:26:42 > 0:26:46But this research rings ethical alarm bells for

0:26:46 > 0:26:52a San Francisco-based society which monitors biotechnology and genetics.

0:26:53 > 0:26:56Once we produce genetically modified human embryos

0:26:56 > 0:26:59in labs around the world, it's really not that

0:26:59 > 0:27:02big of a jump to try to initiate a pregnancy with one of those.

0:27:04 > 0:27:05And for critics,

0:27:05 > 0:27:10it raises the spectre of a brave new world of genetic discrimination.

0:27:11 > 0:27:13You could find wealthy parents

0:27:13 > 0:27:17buying the latest offspring upgrades for their children -

0:27:17 > 0:27:21genetic changes that either did or even that were thought to

0:27:21 > 0:27:24make their children superior in some way.

0:27:24 > 0:27:28And there we could start seeing the emergence of genetic haves

0:27:28 > 0:27:31and have-nots. Some people have called them genetic castes.

0:27:31 > 0:27:32People have thought about this.

0:27:32 > 0:27:35They've called them the GenRich and the Naturals,

0:27:35 > 0:27:39and we could be seeing much greater forms of inequality

0:27:39 > 0:27:43even than the already horrendous levels of inequality we live with.

0:27:43 > 0:27:47Now that the gene genie is out of the bottle,

0:27:47 > 0:27:50society will have to decide what limits should be

0:27:50 > 0:27:55placed on this emerging technology which has the potential to

0:27:55 > 0:28:00alter so much about the world around us and to transform our health.

0:28:02 > 0:28:06Just thinking about the opportunity to cure a genetic disease,

0:28:06 > 0:28:10not treat it, but really provide a cure in the future,

0:28:10 > 0:28:14is so exciting that I think, you know, we want to embrace that

0:28:14 > 0:28:16and we want to enable clinicians

0:28:16 > 0:28:19and scientists to work together to bring that to reality.

0:28:19 > 0:28:23- And do you think diseases will be cured?- I feel they will.

0:28:23 > 0:28:25You know, people say that this is going to be

0:28:25 > 0:28:29the century of biology, and I think there's a lot of truth to that.