With the help of CRISPR, a huge breakthrough in genetic engineering is happening right now: scientists plan to soon learn how to save us forever from any disease, with the prospect of any controlled mutations and eternal life.
We were inspired to publish this post by the video “CRISPR: Gene Editing Will Change Everything Forever”, which talks about the cutting edge of science in terms of human genetic modification: it’s not just about getting rid of diseases like AIDS, cancer and many others, but also about creating a flawless new kind of people, people with superpowers and immortality. And it is happening right now before our very eyes.
All these perspectives are opened up by the recent revolutionary discovery of the protein CRISPR-Cas9, but first things first.
It used to be thought that the DNA in each of our cells is absolutely identical and contains our exact and unchanged copy - no matter which cell we take, but it turned out that this is not the case: the DNA in different cells is slightly different and they change depending on different circumstances.
The discovery of the CRISPR-Cas9 protein was aided by observations of bacterial survivors of viral attacks.
The oldest war on earth
Bacteria and viruses have been competing since the beginning of life: bacteriophage viruses prey on bacteria. In the ocean, they kill 40% of the total number of bacteria every day. A virus does this by inserting its genetic code into a bacterium and using it as a factory.
Bacteria try unsuccessfully to resist, but in most cases they defense mechanisms are too weak. But sometimes bacteria survive. Then they can activate their most effective antiviral system. They store part of the DNA of the virus in their genetic code, the "CRISPR" DNA archive.Here it is stored until the required moment.
When the virus attacks again, the bacterium creates an RNA copy from the DNA archive and
charges the secret weapon - Cas9 protein. This protein scans the bacterium for virus interference, comparing each piece of DNA it finds to the archive. When a 100% match is found, it activates and cuts off the DNA of the virus, rendering it useless, thus protecting the bacterium.
The Cas9 protein scans the DNA of the cell for the introduction of the virus and replaces the damaged part with a healthy fragment.
Tellingly, Cas9 is very precise, like a DNA surgeon. The revolution came when scientists realized that the CRISPR system is programmable—you can just give a copy of the DNA you want to change and put the system in a living cell.
In addition to being accurate, cheap, and easy to use, CRISPR allows you to turn genes on and off in living cells and study specific DNA sequences.
This method also works with any cells, microorganisms, plants, animals or people.
Scientists have found that Cas9 can be programmed for any substitution in any part of the DNA - and this opens up almost limitless possibilities for humanity.
End of sickness?
In 2015, scientists used CRISPR to remove the HIV virus from patients' cells,
and proved that it is possible. A year later, they conducted a more ambitious experiment with rats with the HIV virus in virtually all of their cells.
The scientists simply injected CRISPR into their tails, and they were able to remove over 50% of the virus from cells throughout their bodies. Perhaps in a few decades, CRISPR will help get rid of HIV and other retroviruses - viruses that hide inside human DNA, like herpes. Maybe CRISPR can defeat our worst enemy, cancer.
Cancer is the result of cells that refuse to die and continue to divide while hiding from the immune system along the way. CRISPR gives us the means to edit our immune cells and make them better cancer hunters.
Maybe after a while the cure for cancer will be just a couple of injections with a few thousand of your own cells created in the laboratory to cure you forever.
Perhaps after some time the question of cancer treatment is the question of a couple of injections of modified cells.
The first clinical trial of such therapy in human patients was approved in early 2016 in the United States. Less than a month later, Chinese scientists announced they would be treating lung cancer patients with immune cells modified with the same technology in August 2016. The case is rapidly gaining momentum.
And then there are genetic diseases, thousands of them. They range from mildly annoying to extremely deadly or bring years of suffering. With powerful tools like CRISPR, one day we will be able to do away with this.
Over 3000 genetic diseases caused by a single change in DNA.
We are already creating a modified version of Cas9 that corrects such errors and rids the cell of the disease. In a couple of decades, we may be able to eradicate thousands of diseases forever. However, all these medical applications have one drawback - they are limited to one patient and will die with him if we do not use them on reproductive cells or at an early stage of fetal development.
CRISPR is likely to be used much more widely. For example, to create a modified human, an engineered child. This will bring smooth but irreversible changes in the human gene pool.
Engineered kids
Means of altering the DNA of a human fetus already exist,
but the technology is at an early stage of development. However, it has already been used twice. In 2015 and 2016, Chinese scientists' experiments with human embryos achieved partial success on their second attempt.
They have identified enormous difficulties in gene editing in embryos, but many scientists are already working on solving these problems. It's the same as the computers of the 70s: they will get better in the future.
Regardless of your views on genetic engineering, it will affect everyone. Modified humans can change the genome of our entire species because their grafted traits will be passed on to their children and slowly spread through the generations, slowly changing the human gene pool. It will start gradually.
The first designed children will not be very different from us. Most likely, their genes will be changed to get rid of deadly hereditary diseases.
As technology advances, more people will start to think that not using genetic modification is unethical because it dooms children.
to preventable suffering and death.
As soon as the first such child is born, a door will open that cannot be closed. At first, some traits will be left untouched, but as the technology's acceptance and our knowledge of the genetic code grows, so will the temptation.
If you make your offspring immune to Alzheimer's, why not not give them an improved metabolism? Why not reward them with excellent eyesight to the heap? How about height or muscle? Lush hair? What about the gift of exceptional intelligence for your child?
Enormous changes will come as a result of the accumulation of personal decisions of millions of people.
It's a slippery slope and modified people could be the new normal. As genetic engineering becomes more commonplace and our knowledge improves, we can move towards eradicating the main cause of death, aging.
2/3 of the approximately 150,000 people who have died today have died of aging-related causes.
Today it is believed that aging is caused by the accumulation of damage in our cells.
like DNA breaks or wear and tear on the systems responsible for repairing those damages.
But there are also genes that directly affect our aging.
Genetic engineering and other therapies could stop or slow down aging. Maybe even reverse it.
A typical reaction to the possibility of eternal life (like any other technology that is now familiar, but revolutionary a few hundred years ago).
Eternal Life and the X-Men
We know that there are animals in nature that do not age. Maybe we could borrow a couple of genes from them. Some scientists believe that one day aging will be eradicated. We will still die, but not in a hospital at 90, but after a couple of thousand years, lived surrounded by our loved ones.
The challenge is huge and perhaps the goal is unattainable, but it can be assumed that people living today may be the first to taste the fruits of anti-aging therapy. Perhaps all it takes is convincing a smart billionaire of the need to help solve this big problem.
Looking at it more broadly, we could solve a lot of problems with specially modified people, for example, who could better cope with high-calorie foods, and get rid of such diseases of civilization as obesity.
Owning a modified immune system with a list of potential threats,
we could become immune to most of the diseases that plague us today. Even later, we would be able to create people for long space flights and to adapt to different conditions on other planets, which would be extremely useful for maintaining our life in a hostile universe.
A few pinches of salt
There are several major obstacles, technological and ethical. Many will feel fear of a world where we weed out imperfect people and select offspring based on what is considered healthy.
But we already live in such a world. Testing for dozens of genetic diseases or complications has become the norm for pregnant women in many countries. Often, a single suspicion of a genetic defect can lead to termination of pregnancy.
Take, for example, Down's syndrome, one of the most common genetic defects: in Europe, about 90% of pregnancies diagnosed with this disorder are terminated.
Genetic selection in action: Down syndrome is already diagnosed at an early stage of embryo development and 90% of pregnancies with this diagnosis are terminated.
The decision to terminate a pregnancy is a very personal one, but it's important to understand that we are already selecting people today based on health conditions. There is no point in pretending that this will change, so we need to act carefully and ethically, despite the growing freedom of choice thanks to further development technologies.
However, all these are perspectives of the distant future. Despite the power of CRISPR, the method is not without drawbacks. Editing errors can happen, unknown errors can occur in any part of the DNA and go unnoticed.
Changing the gene can achieve the desired result and cure the disease, but at the same time provoke unwanted changes. We simply do not know enough about the complex relationships of our genes to avoid unpredictable consequences.
Work on accuracy and methods of observation is very important in the upcoming clinical trials. And while we've discussed a possible bright future, a darker vision is also worth mentioning. Imagine what a country like North Korea can do with this level of technology?
It is important that genetic modification technology does not fall into the hands of totalitarian regimes that could hypothetically use it to harm humanity - for example, to create an army of genetically modified soldiers.
Can she extend her reign forever through forced engineering?What will stop a totalitarian regime from creating an army of modified super soldiers?
After all, this is theoretically possible. Scenarios like this are in the distant future, if they are possible at all, but the proof of concept for such engineering already exists. Technology really is that powerful.
This could be a reason to ban engineering and related research, but that would definitely be a mistake. A ban on human genetic engineering will only bring science into a field with rules and laws that we would be uncomfortable with. Only by participating in the process can we be sure that research is conducted with care, reason, control and transparency.
We can research and introduce any genetic modifications into a person.
Conclusion
Feeling anxious? Almost all of us have some kind of imperfection. Would we be allowed to exist in such a new world? Technology is a bit intimidating, but we have a lot to gain, and genetic engineering could be the next step in evolution. intelligent species life.
Perhaps we will end disease, increase life expectancy by centuries, and go to the stars. Do not think small when talking about such a topic. Whatever your opinion about genetic engineering, the future is coming no matter what.
What used to be science fiction will soon become our new reality.
A reality full of possibilities and obstacles.
You can also watch the video itself:
It may seem that DNA is the main center of the molecule, without which its life is impossible. In fact, DNA is a rather sensitive complex molecule, which itself is capable of rapidly changing and exhibiting special properties. It is influenced by both our thoughts and intentions, as well as physical and chemical influences.
Complex chains of genetic codes, each link of which can stop working or become active at any moment - this is what constitutes the concentration of human genetic material. In addition, gene helixes can exhibit incredible properties and help store energy for an incredibly long time. But how is this possible and how can you tune your body to heal by influencing DNA?
light trap
Photons of light are not delayed, but are constantly scattered. In plants, light energy is converted into nutrient molecules, and in the human body, a helical DNA molecule can serve to capture photons of light. This was proven in an experiment with placing DNA in a quartz container and irradiating it with light. Interestingly, the light itself also acquired a spiral structure and could be stored for a month even after the DNA molecule was removed from the container. Such transformation and storage of light energy is available only to helical molecules, which are responsible for the transmission of genetic information.
Self Healing
Many people believe that heredity plays a major role in health. In fact, experimental data on the importance of positive thinking in managing DNA suggests that genes determine us only in part, while the rest of the person is responsible for his own diseases and tendencies. With stress, irritation, constant experiences, genes stop working normally, prerequisites for the development of diseases arise. Pathologies can affect absolutely any organs and tissues, but it all starts with thinking and self-destructive mechanisms of the impact of consciousness on spiral molecules.
The source of energy for the healing of cellular molecules is love. This is a method of targeted healing rejuvenation of cells, preventing their aging and destruction. Love allows you to increase positive energy and make thoughts stronger. Without love, the body cannot develop normally. This is proved by experimental observations, when children cannot fully develop if they lack parental affection and love. It has been proven, for example, that children from shelters are more likely to suffer from autism than babies who are cared for by their parents.
mental transformations
Structural changes in DNA can be influenced at a distance through intention.
If a person consciously concentrates on good thoughts, and his brain begins to radiate harmonious waves, but the DNA helix begins to transform. Moreover, if a person influences with positive thoughts and intentions, then the changes lead to healing transformations, and if there is directed anger, anger, irritation in the thoughts, then the DNA is tuned to the wave of dying. The thing is that the brain begins to transform thoughts into energy flows that are perceived and interpreted by DNA as signals for the restoration of the body, or, conversely, for self-destruction.
According to the experimental data, changes in the structure of DNA placed in an isolated test tube with a neutral medium, in the absence of mental influence, practically did not take place. But when thoughts were focused on the test tube with DNA, changes began in 10% of the sections of the molecule, which carries genetic information. That's how healers work. They are able to convert positive thoughts and attitudes into brain wave energy. It is these waves that give the cells of the body signals about the need to heal organs and systems.
Identical twins have the same set of genes. But for some reason, one does not get out of the disease, and the other never sneezed. It turns out that our health depends not only on what we inherit from our parents, but also on other factors? The science of epigenetics has proven that a person can change what is written for him, that is, his DNA. In what way?
If a person sticks to a balanced diet, forgets about bad habits and acquires good ones, he will not only be able to change his life program, written in his own DNA, but also pass on healthy genes to his descendants, which will extend the years of children and grandchildren.
Garlic turns on the genes
The first and foremost is food. In principle, each of the products can affect the work of genes. But there are some, the usefulness of which scientists have already proven 100 percent.
Among them - green tea. Green tea contains catechins (epigallocatechin-3-gallate, epicatechin, epicatechin-3-gallate, epigallocatechin), which can suppress cancer-causing genes and activate those genes that can fight tumors. Drinking 2-3 small cups of green tea every day is enough to keep your DNA in anti-cancer combat readiness. Green tea is especially useful for women, among whose relatives there are patients with breast tumors.
Another product is garlic. Other compounds work in garlic - diallyl sulfide, diallyl disulfide, diallyl trisulfide. It is necessary to eat 2-3 cloves of garlic a day in order to start the genes that manage not only the processes of death of cells that give metastases, but also fight old age, prolong life.
The third panacea is soy. Soy contains isoflavones (genistein, daidzein) - an effective antitumor agent for breast cancer, prostate, larynx, colon and leukemia. Scientists advise using soy in dietary supplements and sticking to the dosage indicated on the packages.
The fourth fighter for healthy genes is grapes and products from it (juice and wine). A bunch of dark grapes (that's 120 g of grape juice or 100 g of dry red wine) added to your daily menu will provide the body with resveratrol, a gene-changing substance.
In a diet that will appeal to good genes, it is worth including 100 g of dark red tomatoes (substance lycopene) with the addition of olive oil. Tomatoes should be eaten four times more if there are cancer patients in the family.
Another vegetable that your heirs will remember with a kind word is broccoli (substance indole-3-carbinol). 100 g of broccoli - each, 300 g - at risk of cancer.
Be sure to eat nuts, fish, eggs and mushrooms - they provide the body with microelements selenium and zinc, which also change DNA.
The obese constitution was fixed in the genome
The work of genes depends on the diet. The diet should be low-calorie (no more than 2 thousand kcal per day). It delays the aging of a person, guarantees longevity to his children and grandchildren. Epigenetics also explains the obesity epidemic that has broken out today: we are becoming more and more full because our mothers overeat before and during pregnancy. This is confirmed by experiments conducted on animals: overfed mice each time produced even more obese offspring, and a similar constitution was fixed in the genome.
Genes like it when their master keeps himself in good shape. physical form. Scientists have determined that regular exercise for 45 days on a regular exercise bike activates about 500 genes! And if you practice regularly and further, you can change even more genes for the better.
About bad habits written-rewritten. But the influence of cigarettes, alcohol and drugs directly on genes has only recently been proven. It turns out that more than 150 sections of DNA in chronic alcoholics get abnormal activity. Result: the alcoholic cannot concentrate, does not remember anything, cannot control his emotions. But the saddest thing is that he passes on diseased genes to offspring.
And about 120 genes remain changed even 10 years after quitting cigarettes. And again, among them are the most important genes that control cell division. The result is cancer in the smoker. But there is reason for optimism: genes can be corrected, and the less experience of addiction to, the sooner this can be done.
Genes are also affected by emotions, both positive and negative, received at home, in the family, at work.
And, finally, the ecological situation in which a person lives. Obviously, industrial emissions, car exhausts, nitrates in food, polluted water also lead to breakdowns in the genes.
Do you want to live longer? Do you wish health to your children and grandchildren? Then take care of your genes.
Now you know how to do it?
Changing human DNA that is passed on to future generations has long been considered ethically closed and banned in many countries. Scientists report they are using new tools to repair disease genes in human embryos. Although the researchers are using defective embryos and have no intention of implanting them in a woman's uterus, the work is troubling.
A change in the DNA of human eggs, sperm, or embryos is known as a germline change. Many scientists are calling for a moratorium on the revision of clinical embryos, editing the human germ line, and many believe that this type of scientific activity should be banned.
However, editing the DNA of a human embryo may be ethically acceptable to prevent illness in a child, but only in rare cases and with guarantees. These situations can be limited to couples where they both have serious genetic conditions and for whom embryo editing is really the last reasonable option if they want to have a healthy baby.
The danger of deliberately changing genes
Scientists believe that editing a human embryo may be acceptable to prevent a child from inheriting serious genetic diseases, but only if certain safety and ethical criteria are met. For example, a couple cannot have "reasonable alternatives" such as being able to select healthy embryos for in vitro fertilization (IVF) or through prenatal testing and abortion of a fetus with a disease. Another situation that may meet the criteria is if both parents have the same medical condition, such as cystic fibrosis.
The scientists warn of the need for strict government oversight to prevent germline editing from being used for other purposes, such as giving a child desirable, distinctive traits.
By editing genes in patient cells that are not inherited, clinical trials are already underway to fight HIV, hemophilia and leukemia. It is believed that the existing regulatory systems for gene therapy are sufficient to carry out such work.
Genome editing should not be to increase potency, increase muscle strength in a healthy person, or lower cholesterol levels.
Human germline gene editing or human germline modification means the intentional modification of genes that is passed on to children and future generations.
In other words, creation of genetically modified humans. Human germline modification has been considered a taboo subject for many years due to safety and social reasons. It is formally banned in more than 40 countries.
Experiments on the creation of genetically modified people and the science of eugenics
However, in last years, according to new methods of genetic engineering, experiments were carried out with human embryos. For research, genes and human embryos associated with beta blood disease - thalassemia were used. The experiments were mostly unsuccessful. But gene-editing tools are being developed in labs around the world and are expected to make it easier, cheaper, and more accurate to edit or delete genes than ever before. Modern yet theoretical methods of editing the genome will allow scientists to insert, delete and tweak DNA with positive results. This holds the promise of treating certain diseases, such as sickle cell disease, cystic fibrosis, and certain types of cancer.
Selection in relation to humans - eugenics
Gene editing of human embryos or the direction of eugenics leads to the creation of genetically modified very different people. This causes serious security due to social and ethical issues. They range from the prospect of irreversible harm to the health of future children and generations, to opening doors to new forms of social inequality, discrimination and conflict and a new era of eugenics.
The science of eugenics for human selection came into existence in the middle of the last century as a science of the Nazi direction.
Scientists are not allowed to make changes to human DNA, which is passed on to subsequent generations. Such an innovative step in the science of eugenics should be considered only after additional research, after which changes can be made under severe restrictions. Such work should be prohibited in order to prevent serious illness and disability.
Variation caused by changing genes is also called mutations.
It is a long taboo against making changes in the genes of human sperm, eggs or embryos, because such changes will be inherited by future generations. This is taboo in part because of the fear that mistakes could inadvertently create new artificial diseases that could then become a permanent part of the human gene pool.
Another problem is that this species can be used for genetic modification for non-medical reasons. For example, scientists could theoretically try to create a child constructor in which parents try to select the traits of their children to make them smarter, taller, better athletes, or with other supposedly necessary attributes.
Nothing like this is currently possible. But even the prospect causes fears of scientists to significantly change the course of evolution and the creation of people who are considered genetically improved, to come up with what dystopias of the future, described in films and books.
Any attempt to create babies from sperm, eggs or embryos that have their own DNA and attempt to edit can only be done under very carefully controlled conditions and only to prevent devastating disease.
It can be difficult to further distinguish between using gene editing to prevent or treat a disease and using it to enhance a person's abilities.
For example, if scientists manage to find out that gene changes increase mental abilities to fight off dementia in Alzheimer's disease, then this can be considered preventive medicine. If you simply radically improve the memory of a healthy person, then this is no longer a medical direction.
When is it allowed to change DNA
The ability to edit genes can be used to treat many diseases and possibly even prevent many devastating disorders from occurring in the first place by editing out genetic mutations in sperm, egg and embryo. Some potential changes could prevent a wide range of diseases, including breast cancer, Tay-Sachs disease, sickle cell anemia, cystic fibrosis, and Huntington's disease.
Clinical trials for gene editing should be allowed if:
- no “reasonable alternative” to prevent “serious illness”
- it has been convincingly proven that genes, when edited, eliminate the cause of the disease
- changes are aimed only at the transformation of such genes that are associated with the usual state of health
- sufficient preliminary research work on risks and potential health benefits
- continuous, rigorous supervision to study the effect of the procedure on the health and safety of participants, and long-term comprehensive plans
- there is maximum transparency in accordance with patient confidentiality and reassessment of health, social benefits and risks is underway
- there are robust oversight mechanisms in place to prevent the spread of a serious illness or condition.
Proponents of human germline editing argue that it could potentially reduce, or even eliminate, the occurrence of many serious genetic diseases that would reduce human suffering throughout the world. Opponents say that altering human embryos is dangerous and unnatural, and does not take into account the consent of future generations.
Discussion on the change of the human embryo
Let's start with the objection that changing the fetus is unnatural or playing against God.
This argument is based on the premise that natural is inherently good.
But illnesses are natural and people by the millions fall ill and die prematurely - all quite naturally. If we only protected natural beings and natural phenomena, we would not be able to use antibiotics to kill bacteria or otherwise practice medicine or fight drought, famine, pestilence. The health care system is maintained in every developed country and can rightly be described as part of a comprehensive attempt to thwart the course of nature. Which of course is neither good nor bad. Natural substances or natural treatments are better, if they are, of course, possible.
Leads to an important moment in the history of medicine and genome editing and represents promising scientific endeavors for the benefit of all mankind.
Interference with the human genome is permitted only for prophylactic, diagnostic or therapeutic purposes and without modification for offspring.
Rapid progress in the field of genetics, the so-called "designer babies" increases the need for bioethics for a broader public and debate about the power of science. Science is able to genetically modify human embryos in the laboratory to control inherited traits such as appearance and intellect.
As of now, many countries have signed an international convention banning this kind of gene editing and DNA modification.
Jennifer Doudna is a well-known scientist from the USA, whose works are mainly devoted to structural biology and biochemistry. Jennifer is a laureate of many prestigious awards, in 1985 she received a bachelor's degree, and already in 89 she became a Ph.D. Harvard University. Since 2002 he has been working at the University of California at Berkeley. She is widely known as a researcher of RNA interference and CRISPR. Conducted research on Cas9 with Emmanuelle Charpentier.
00:12
A few years ago, my colleague Emmanuelle Charpentier and I invented a new technology for editing genomes. It's called CRISPR-Cas9. CRISPR technology allows scientists to make changes to the DNA inside cells, which could enable us to cure genetic diseases.
00:31
You may be interested to know that CRISPR technology originated as part of a basic research project to understand how bacteria fight viral infections. Bacteria have to deal with viruses in their environment, and a viral infection can be thought of as a ticking time bomb: the bacterium has only a few minutes to render it harmless before the bacterium is destroyed. In the cells of many bacteria there is an adaptive the immune system- CRISPR, allowing them to detect and destroy viral DNA.
01:04
The CRISPR system includes the Cas9 protein, which is able to search for, cleave, and ultimately destroy viral DNA in a special way. And it was during our research into the activity of this protein, Cas9, that we realized that we could use its activity in a genetically engineered technology that would allow scientists to remove and insert DNA fragments inside cells with incredible precision, which would allow us to do what was previously it was simply impossible.
01:42
CRISPR technology is already being used to change DNA in mice and monkeys, as well as other organisms. Recently, Chinese scientists have shown that they have even been able to use CRISPR technology to modify the genes of human embryos. Scientists from Philadelphia have shown the possibility of using CRISPR to remove the DNA of an integrated HIV virus from infected human cells.
02:09
The ability to do genome editing in this way also raises various ethical issues to keep in mind, because the technology can be applied not only to adult cells, but also to embryos. different organisms, including our species. Thus, together with colleagues, we began an international discussion of the technology we invented in order to be able to take into account all the ethical and social problems associated with such technologies.
02:39
And now I want to tell you what CRISPR technology is, what it allows you to do, where we are now and why I think that we need to move forward with caution on the path of using this technology.
02:54
When viruses infect a cell, they inject their DNA. And inside the bacterium, the CRISPR system allows you to pull this DNA out of the virus and insert small fragments of it into the chromosome - into the DNA of the bacterium. And these pieces of viral DNA are inserted into a region called CRISPR. CRISPR stands for "short palindromic repeats regularly arranged in clusters". (Laugh)
03:24
Too long. Now you understand why we use the acronym CRISPR. This is a mechanism that allows cells to register, over time, the viruses that infect them. And it is important to note that these DNA fragments are passed on to the descendants of cells, so that cells are protected from viruses not for one generation, but for many generations of cells. This allows the cells to keep a "record" of the infection, and as my colleague Blake Widenheft says, the CRISPR locus is actually the cell's genetic vaccination card. After inserting these DNA fragments into the bacterial chromosome, the cell makes a small copy in the form of a molecule called RNA, in this picture it is orange, and this is an exact print of the viral DNA. RNA is the chemical "cousin" of DNA, which allows it to interact with DNA molecules that have a suitable sequence for it.
04:24
So these little pieces of RNA from the CRISPR locus associate, bind to a protein called Cas9, which is white in this picture, and form a complex that acts as a sentry in the cell. It scans all of the DNA in the cell to find regions that match the RNA sequences associated with it. And when these sites are found, as you can see in the figure, where DNA is the blue molecule, this complex binds to this DNA and allows the Cas9 protein to cut the viral DNA. He cuts the gap very accurately. We can think of this sentry, the complex of the Cas9 protein and RNA, as a pair of scissors that can cut DNA - it makes a double-strand break in the DNA helix. And it is important that this complex can be programmed, for example, it can be programmed to recognize the necessary DNA sequences and cut the DNA in this area.
05:26
As I'm about to tell you, we realized that this activity could be used in genetic engineering to allow cells to make very precise changes to the DNA at the site where the cut was made. It's like using a word processing program to correct typos in a document.
05:48
We were able to suggest that the CRISPR system can be used in genomic engineering, since cells are able to find broken DNA and repair it. So, when a plant or animal cell finds a double-strand break in its DNA, it is able to repair it, either by joining the broken ends of the DNA, making a slight change in the sequence at that location, or it can repair the break by inserting a new stretch of DNA at the break. Thus, if we can introduce double-strand breaks in DNA in strictly defined places, we can force cells to repair these breaks, while either destroying genetic information or introducing new one. And if we could program the CRISPR technology so that a DNA break is introduced at or near a mutation that causes cystic fibrosis, for example, we could get cells to fix that mutation.
06:51
Actually, genomic engineering is not a new field, it has been developing since the 1970s. We have the technology to sequence DNA, to copy DNA, even to manipulate DNA. And these are very promising technologies, but the problem is that they were either ineffective or too difficult to use, so most scientists could not use them in their laboratories or apply them in clinical settings. So there was a need for a technology like CRISPR because it's relatively easy to use. You can think of old genome engineering as having to rewire your computer every time you want to run a new program, whereas CRISPR is something like software for the genome: we can easily program it using small pieces of RNA.
07:53
Once a double-strand break is made, we can trigger a repair process and thereby possibly achieve amazing results, such as fixing mutations that cause sickle cell anemia or Huntington's disease. Personally, I believe that the first applications of CRISPR technology will be in the blood, where it is relatively easy to deliver this tool into cells, compared to dense tissues.
08:22
Right now, a lot of ongoing work is using the method in animal models of human disease, such as mice. Technology is used to make very precise changes, which allows us to study how these changes in cellular DNA affect either tissue or, as here, the whole organism.
08:42
In this example, CRISPR technology was used to disrupt a gene by making a small change to the DNA in the gene responsible for the black coat color in these mice. Imagine, these white mice differ from their colored brothers and sisters in just a small change in one gene in the entire genome, but otherwise they are completely normal. And when we sequence the DNA of these animals, we find that the change in the DNA happened exactly in the place where we planned, using CRISPR technology.
09:18
Experiments are also being carried out on other animals in which it is convenient to create models of human diseases, for example, on monkeys. And in this case, we find that these systems can be used to test the application of this technology to certain tissues, for example, to figure out how to deliver a CRISPR tool into cells. We also want to expand our understanding of how we can control how DNA is repaired after it breaks, and find out how we can control and limit off-target effects, or unintended effects, when using this technology.
09:55
I believe that we will see the use of this technology in the clinic, of course, in adult patients, within the next 10 years. It seems likely to me that there will be clinical trials and perhaps even therapies approved during this period, which is very encouraging. And because of this excitement that the technology generates, there is a huge interest in it from start-up companies created to turn CRISPR technology into a commercial product, as well as many venture capitalists,
10:26
investing in such companies. But we must also consider that CRISPR technology can be used to improve performance. Imagine if we could try designing humans with improved characteristics, such as stronger bones, or less propensity for cardiovascular disease, or even features that we might think would be desirable, such as a different eye color or more high growth, something like that. If you want, these are "design people". Now there is practically no genetic information that allows us to understand which genes are responsible for these traits. But it is important to understand that CRISPR technology has given us the tool to make these changes,
11:13
as soon as this knowledge becomes available to us. This raises a number of ethical questions that we must carefully consider. And that is why my colleagues and I called on scientists around the world to pause any clinical applications of CRISPR technology in human embryos so that we have time to carefully consider all possible consequences this. And we have an important precedent for such a pause: in the 1970s, scientists came together to declare a moratorium on the use of molecular cloning,
11:47
until the technology has been thoroughly tested and proven safe. So while the genetic engineering of humans is delayed, it is no longer science fiction. Genetically engineered animals and plants already exist. And this imposes on all of us a great responsibility and the need to consider both the undesirable consequences and the role of the deliberate influence of this scientific breakthrough.
12:21
Thank you!
12:22
(Applause) (Applause ended)
Bruno Giussani: Jennifer, this technology could have huge implications, as you pointed out. We highly respect your position on the announcement of a pause, or a moratorium, or a quarantine. All of this, of course, has therapeutic implications, but there are also non-treatment ones, and it seems that these are the ones that attract the most interest, especially in the media. Here is one of the latest issues of The Economist: "Editing Humanity." It only talks about improving properties, not about healing. What kind of reaction did you get in March from your colleagues in the scientific community when you asked or offered to pause and think about all this?
Jennifer Doudna: I think colleagues were glad to have the opportunity to discuss this openly. It is interesting that when I talked about this with people, my fellow scientists and not only expressed very different points of view on this matter. Obviously, this topic requires careful consideration and discussion.
BJ: There will be a big meeting in December that you and your colleagues are convening together with the National Academy of Sciences and others. What exactly do you expect from this meeting, from a practical point of view?
JD A: I hope that the views of many people and stakeholders willing to responsibly consider the use of this technology will be made public. It may not be possible to reach a consensus, but I believe that we should at least understand what problems we will face in the future.
BJ: Your colleagues, for example, George Church at Harvard, say: “Ethical issues are basically a security issue. We test again and again on animals, in laboratories, and when we feel that there is no danger, we switch to humans.” This is a different approach: we must seize this opportunity and must not stop. Could this cause a split in the scientific community? That is, we will see that some people will retreat because they doubt ethics, while others will simply go forward, since in some countries there is little or no control.
JD : I think any new technology, especially one like this, will have a few different points of view, and I think that's completely understandable. I believe that eventually this technology will be used to construct the human genome, but it seems to me that doing this without carefully considering and discussing the risks and possible complications it would be irresponsible.
BJ: There are many technologies and other fields of science that are developing exponentially, in fact, as in your field. I mean artificial intelligence, autonomous robots and so on. Nowhere, it seems to me, except for the field of autonomous military robots, has anyone initiated a similar discussion in these areas, calling for a moratorium. Do you think that your discussion can be an example for other areas?
JD: I think it's hard for scientists to leave the lab. Speaking about me, I'm not very comfortable doing it. But I do believe that since I am involved in the development of this, this fact imposes a responsibility on me and my colleagues. And I would say that I hope that other technologies will be considered in the same way that we would like to consider something that can have an impact. in fields other than biology.
15:44
BJ: Jennifer, thank you for coming to TED.
JD: Thanks!
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