CRISPR: What is the future of gene editing? | Start Here
Summary
TLDRCRISPR-Cas9, a revolutionary gene-editing technology, offers unprecedented potential to combat genetic diseases like muscular dystrophy and cancer. Discovered by Emmanuelle Charpentier and Jennifer Doudna, it functions as 'genetic scissors' by targeting and replacing faulty genes with healthy ones. However, ethical concerns arise with germ-line editing, as seen in the controversial case of the world's first CRISPR-modified babies by He Jiankui, which sparked international outrage and calls for stricter regulations. While 75 countries ban its use in reproduction, the technology's potential to transform lives remains immense, despite the risks and unknowns.
Takeaways
- đŹ CRISPR is a revolutionary gene-editing technology that has been around for less than a decade and has already been recognized with a Nobel Prize in 2020.
- 𧏠Genes are the body's blueprint, made up of DNA that instructs cells to make proteins, which in turn determine our physical traits and functions.
- đ ïž CRISPR-Cas9 acts as a pair of 'genetic scissors', allowing scientists to cut out faulty genes and potentially replace them with healthy ones.
- đ The discovery of CRISPR was accidental, stemming from research into how bacteria use CRISPR to store and utilize pieces of viral DNA for defense.
- đ CRISPR has been used in medical treatments, such as for sickle cell disease, with one patient, Victoria Gray, experiencing significant symptom reduction.
- đ« The use of CRISPR on germ-line cells, which can affect future generations, is controversial and largely prohibited due to ethical and safety concerns.
- đ¶ The birth of the world's first CRISPR-modified babies by Chinese scientist He Jiankui in 2018 sparked international outrage and raised questions about consent and the unknown long-term effects.
- đą He Jiankui faced legal consequences for his unauthorized experiments, receiving a three-year prison sentence and a lifetime ban from reproductive medicine.
- đ± Despite the controversy, CRISPR research continues in other areas, such as plants and animals, indicating its broad potential applications.
- đ There is a push for more regulation and guidelines regarding the use of CRISPR to ensure ethical practices and prevent misuse.
- đ The script highlights the dual nature of CRISPR's potential, emphasizing both its capacity to transform lives positively and the risks associated with its misuse.
Q & A
What is CRISPR and how does it relate to gene editing?
-CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats, is a revolutionary gene-editing technology that allows scientists to perform precise changes to the DNA within living organisms. It functions as a pair of genetic scissors, enabling the targeting and editing of specific genes.
Who were the scientists awarded the Nobel Prize for the development of CRISPR?
-Emmanuelle Charpentier and Jennifer Doudna were awarded the Nobel Prize in Chemistry in 2020 for their work on the CRISPR-Cas9 genetic scissors.
How does CRISPR-Cas9 work in the context of bacteria?
-In bacteria, CRISPR-Cas9 works as a defense mechanism against viruses. It stores snippets of viral DNA, and when the virus returns, the CRISPR DNA is replicated. Parts of this replication join with the Cas9 protein to locate and cut the viral DNA, thereby neutralizing the threat.
What is the potential application of CRISPR in treating diseases?
-CRISPR has the potential to treat, and possibly cure, genetic diseases such as muscular dystrophy, cystic fibrosis, and certain types of cancer by editing faulty genes and replacing them with healthy ones.
Can you explain the case of Victoria Gray and how CRISPR was used to treat her?
-Victoria Gray was the first person in the US to receive a CRISPR-based treatment for sickle cell disease. Doctors infused her with over 2 billion of her own genetically edited bone marrow cells, which helped to alleviate her symptoms significantly.
What is the difference between editing somatic cells and germ-line cells in the context of CRISPR?
-Somatic cells are all the cells in the body except for sperm and egg cells. Genetic changes made in somatic cells are not passed on to offspring. In contrast, germ-line cells are sperm, egg, or fertilized eggs, and changes to these cells can be inherited by future generations.
Why was the use of CRISPR by He Jiankui controversial?
-He Jiankui's use of CRISPR was controversial because he edited germ-line cells to create the world's first CRISPR-modified babies, which raised ethical concerns about consent, the potential for unintended genetic mutations, and the societal implications of designer babies.
What are the current regulations regarding the use of CRISPR in human reproduction?
-Seventy-five countries have prohibited the use of CRISPR in human reproduction. There is an ongoing effort by scientists and governments to establish more comprehensive regulations and guidelines to govern the use of this technology.
What was the outcome for He Jiankui after his controversial use of CRISPR?
-He Jiankui was sentenced to three years in prison and was banned from working on reproductive medicine for life due to his unauthorized and ethically questionable use of CRISPR technology on human embryos.
How is CRISPR being used in non-human applications?
-CRISPR is being experimented with in plants and animals for various purposes, such as improving crop yields, enhancing disease resistance, and studying animal genetics, without the ethical concerns associated with human germ-line editing.
What are the potential risks of using CRISPR technology?
-The potential risks of using CRISPR include unintended genetic mutations with unpredictable side effects, the ethical issue of consent (especially in the case of embryos), and the broader societal implications of altering the human gene pool.
Outlines
𧏠CRISPR: Revolutionizing Gene Editing
The script introduces CRISPR, a groundbreaking gene-editing technology that has been acknowledged with a Nobel Prize. It discusses the potential of CRISPR to combat diseases and enhance crops, while also touching on ethical concerns and fears. The summary explains how CRISPR-Cas9 functions as 'genetic scissors,' enabling scientists to edit faulty genes, with an example of its application in treating sickle cell disease. The segment also raises questions about the extent to which gene editing should be applied, especially concerning germ-line editing and the controversial case of the world's first CRISPR-modified babies.
đ« Ethical and Regulatory Challenges of CRISPR
This paragraph delves into the ethical implications and regulatory responses to CRISPR technology. It highlights the permanent and hereditary nature of germ-line cell editing, the unpredictable side effects of genome mutations, and issues of consent, especially in the case of the CRISPR-modified babies born to HIV-resistant parents without their consent. The summary outlines the international efforts to draft guidelines for CRISPR use and mentions the legal consequences faced by the Chinese scientist He Jiankui. It concludes with a reflection on the dual nature of CRISPR's potential to improve lives while also posing significant risks if misused.
Mindmap
Keywords
đĄGene editing
đĄCRISPR
đĄNobel Prize
đĄDNA
đĄProteins
đĄGenetic diseases
đĄSomatic cells
đĄGerm-line cells
đĄDesigner babies
đĄRegulation
đĄCRISPR-Cas9
Highlights
CRISPR is a revolutionary gene editing technology less than 10 years old that can perform surgery on genes.
CRISPR inventors Emmanuelle Charpentier and Jennifer Doudna won the Nobel Prize in 2020.
CRISPR can potentially fight diseases and improve crops.
Genes are the blueprint for making everything the body needs, with over 20,000 in humans.
Some gene variations can cause sickness, but CRISPR may enable scientists to change those genes.
CRISPR-Cas9 acts as a pair of genetic scissors, allowing targeted gene editing.
Charpentier and Doudna discovered CRISPR by studying how bacteria protect themselves from viruses.
CRISPR is simple to use, cheap, and has the potential to treat or cure genetic diseases like muscular dystrophy, cystic fibrosis, and cancer.
Victoria Gray was the first person in the US to receive a CRISPR-based treatment for sickle cell disease, with significant symptom improvement.
CRISPR used in this way is not hereditary and does not affect the human gene pool.
Some suggest using CRISPR on early embryos for genetic fixes, but many scientists consider this a step too far due to safety and societal concerns.
Chinese scientist He Jiankui caused outrage by creating the world's first CRISPR-modified babies, altering their germ-line cells.
He Jiankui was sentenced to three years in prison and banned from reproductive medicine for life.
The potential misuse of CRISPR raises concerns about creating designer babies and permanently altering the human gene pool.
Seventy-five countries prohibit the use of CRISPR in human reproduction, but more regulations are needed.
An international commission is drafting guidelines to regulate the use of CRISPR.
CRISPR research continues in plants and animals, despite the controversy surrounding its use in humans.
The full potential of CRISPR to transform lives without harming others is a power that people are unlikely to ignore.
Transcripts
Letâs talk about gene editing and its potential to change lives.
Itâs because of CRISPR â a technology thatâs not even 10
years old and can basically perform surgery on our genes.
The inventors got a Nobel Prize for it in 2020.
CRISPR fans say itâll fight diseases and improve crops.
Other people? Well, Hollywood tapped into their fears a long time ago.
At least one scientist has crossed the line.
So how does gene editing actually work?
Can CRISPR be used to cure disease?
And how far should we be allowed to go in transforming the human race?
Genes are the blueprint for making everything the body needs.
And human beings have more than 20,000 of them.
Genes are made up of DNA and that instructs the cell how to make a protein.
Those proteins carry out the functions of the body and determine the way we look.
But some gene variations can make us sick.
Scientists, though, may now have the unprecedented power to change those genes.
Itâs all because of the work done by two Nobel Prize winning scientists:
Emmanuelle Charpentier and Jennifer Doudna.
What they discovered has unleashed a whole new era of genetic engineering
thatâs advancing just about every day.
The tool is called CRISPRâCas9.
And like so often happens in science they discovered it by chance.
Charpentier and Doudna were studying how bacteria protect themselves from viruses
because, yes, bacteria get sick too.
They found that bacteria have a part of their DNA called CRISPR
that stores tiny pieces of a virusâs DNA.
A copy of that bacterial CRISPR DNA is made the next time the virus comes back.
Small parts of the copy then join up with a protein called Cas9.
So CRISPRâCas9 goes looking for that matching bit of DNA
in the virus, latches onto it and through a chemical reaction
it snips the viral DNA to destroy it.
Itâs why scientists describe CRISPRâCas9 as a pair of genetic scissors.
So Charpentier and Doudna then thought:
Why canât we harness that same natural process
but programme it to cut out genes that are faulty
and replace them with healthy ones.
That was the lightbulb moment.
Itâs simple to use and cheap and that makes it revolutionary.
Because it could treat, perhaps even cure, genetic diseases
like muscular dystrophy, cystic fibrosis, even cancer.
And a lot of attention has been on sickle cell disease.
In fact scientists have already used it on a woman named Victoria Gray.
She was the first person in the US to receive a CRISPR-based treatment for the disease.
Doctors infused her with more than 2 billion of her own genetically edited bone marrow cells.
More than a year later almost all her symptoms are gone.
Just to be clear â CRISPR is being used here like any other medical treatment.
Any genetic changes stay with that patient. They arenât passed on to their children.
Theyâre not hereditary changes that affect the human gene pool forever.
Thatâs because scientists are fixing faulty genes in somatic cells â basically all the cells in
our bodies except the single-cell sperm and egg.
And itâs important to point out the patient is an adult whoâs consented to the treatment.
But some are suggesting using CRISPR on early embryos to make the genetic fix.
And for a lot of safety and societal reasons scientists say thatâs a step too far.
But that didnât stop a Chinese scientist from going there.
In 2018 He Jiankui announced the birth of the worldâs first CRISPR-modified babies â
twin girls known only as Lulu and Nana.
There was outrage.
Heâd altered a gene to try to protect them from HIV because their father has AIDS.
But he did it while the girls were still in a petri dish.
And thatâs why his use of CRISPR technology is a problem.
He genetically altered what are called germ-line cells:
the sperm, the egg, or in the case of the twin girls, a fertilised egg at the single-cell stage.
Changes to DNA here appear in every cell in a personâs body.
You might say thatâs an efficient fix
but those genetic changes are permanent and are passed onto future generations.
Plus, this is unchartered territory.
Scientists say thereâs a very real risk that an edit could cause a mutation in the genome with side effects that we just canât predict.
Thereâs also the issue of consent.
The girls obviously had no say in the changes made to their bodies.
That experiment alone raised the spectre of designer babies, making people smarter, stronger, maybe more attractive.
So whatâs being done to regulate it?
Seventy-five countries already prohibit the use of CRISPR in human reproduction.
But many scientists and governments agree more rules are needed.
Right now we have an international commission thatâs started to draft some guidelines.
None of that, though, has stopped scientists from experimenting with CRISPR â
in plants and animals at least.
So what happened to the man who did interfere?
Well, He Jiankui was sentenced to three years in prison and banned from working on reproductive medicine for life.
And the twin girls? No one really knows where they are or whether theyâre healthy.
We donât even know who else in the world is trying this technology.
Thereâs a good chance someone will misuse it and potentially change the human race.
Thatâs enough to want to bury this technology.
But CRISPR has enormous potential to transform some peopleâs lives without hurting anyone else.
Thatâs a power people just wonât want to ignore.
If you want to get a better understanding of the stories in the news, you can find all of our episodes on YouTube.
Just look for Start Here.
You can also find us on Al Jazeeraâs Facebook page, Instagram and on our website.
Weâll see you next week.
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