I can extract side effects

During this time, Cao Yang has been following up with Tang Que on the gene therapy.

The two parties reached a consensus that gene editing tools should be used to completely knock out the extra A bases on the ATP7A gene, so as to fundamentally treat Menkes patients.

However, there are different opinions on which gene editing tool to choose.

After entering the 21st century, with the development of biotechnology, scientists continue to develop in order to realize gene editing. Up to now, three generations of new technologies have been produced, namely ZFN (zinc finger nucleoproteinase), TALENs (transcription activator-like effector Nuclease) and CRISPR-Cas9 are three gene editing tools.

ZFN zinc finger protein technology is the first generation of gene editing tools and has been available for more than 20 years.

It can recognize and bind a specific triplet base through the zinc finger protein widely present in eukaryotes (from yeast to human), so as to achieve the purpose of DNA site-directed shearing.

TALEN technology is the second-generation gene editing tool that appeared ten years ago. It has achieved considerable breakthroughs in technology. It can complete gene editing work in a more efficient way, and it is rare to accidentally cut off non-target DNA sequences. So far So far, it has received high praise.

TALENs use a natural protein secreted by plant bacteria to recognize specific DNA base pairs, bind to target DNA and cut DNA strands at specific sites, thereby introducing new genetic material.

Compared with ZFNs, TALENs can target longer gene sequences and are easier to construct.

But until now, there has been no low-cost, publicly available method to rapidly generate large quantities of TALENs.

CRISPR-Cas9 is the third generation and the latest gene editing tool. It was invented by two female scientists and won the Nobel Prize last year.

This is a breakthrough new technology, which is fundamentally different from the first and second generation genome editing technology, so it is often called "gene magic scissors". global popularity.

CRISPR was originally a natural immune system invented by bacteria during the evolution of billions of years ago. After some bacteria are invaded by viruses, they can store a small segment of the virus gene in a storage space called CRISPR in their own DNA. When the virus invades again, once the bacteria finds the same part as the stored fragment, the Cas9 protein will deform, accurately clamp the viral DNA and swing the "scissors" without hesitation.

CRISPR can make changes or mutations in the genome more efficiently than other gene-editing techniques such as TALEN.

It finds the DNA that needs to be cut as the target through the guide RNA, and then uses Cas9, an enzyme specially used for cutting DNA, to operate, thereby precisely cutting the DNA target and realizing a series of gene editing such as insertion, knockout, silencing, and replacement. operate.

The reason why this technology has attracted so much attention is that it can precisely manipulate genes in an extremely simple way.

Simple and convenient, easy to use, even an undergraduate can do it, and the price is low, so it instantly became popular all over the world.

Although the hottest gene editing technology is CRISP-Cas9, the three editing systems actually have their own advantages.

It can only be said that each researcher needs to combine his own topic in order to choose the most suitable tool.

With these gene editing tools, major pharmaceutical companies and biotechnology companies around the world have successively developed a series of clinical trial drugs in the field of rare diseases.

This is mainly because rare diseases are monogenic hereditary diseases, which can be treated by modifying genes.

And those more common diseases, including chronic diseases such as cancer and high blood pressure, cannot be cured even by modifying dozens of genes at present, and their pathogenic factors are very complicated.

Out of caution, Tang Que did not want to adopt the latest CRISP-Cas9 tool.

Firstly, there is currently no gene drug that has been successfully developed using this tool in the world. According to relevant research, its off-target rate will be relatively high, which will lead to the failure of drug development.

Secondly, its underlying patents belong to Eagle Country. If it is to be applied commercially, it will involve the issue of patent licensing. Business negotiations in this area will take a long time, and patients may not be able to wait.

Among the three current gene editing tools, only ZFN's patent has expired, and there is no such problem.

If Cao Yang personally uses CRISP-Cas9 for development, there is no problem. The patentee does not restrict the use of scientific research, and his research and efforts all the time are also aimed at this tool.

However, as a commercial pharmaceutical company, Sanqing has more considerations and is more inclined to choose tools that are not troubled by patents.

However, the problem is not big. After friendly negotiation, the two parties decided to use the ZFN gene editing tool for gene knockout.

The precision of ZFN approaches is unpredictable because of the interplay of DNA-binding elements, and therefore, proteins that map to each DNA sequence need to be constructed and tested.

This undoubtedly requires a high degree of expertise and is a lengthy and expensive verification process.

Fortunately, Sanqing has AICell as a good helper, which can greatly speed up the work in this area.

During this period, another seven or eight Menkes patients were brought to Kunming by their family members, where they underwent detailed tests on Sanqing's gene sequencer to find gene targets and carry out targeted preparatory work.

The entire laboratory is like a machine running at full speed, running at full speed.

*****

Time passed quickly, and Wei Kang waited for the Nobel Prize Ceremony in December.

Before that, he posted a message on Langbo, stating that he was busy with the great cause of gene therapy and had no time to go to Stockholm to attend the ceremony, so he sent a company employee to collect it on his behalf.

The news sparked a heated discussion in an instant.

Netizens have expressed their understanding.

"Mr. Wei is now a national scholar of Huaxia. It is a strategic material. He must not go abroad, otherwise he will definitely be detained by the shameless Eagle Kingdom."

"That's right, Yingguo is so despicable and shameless. It is strongly recommended that Mr. Wei stay in the company every day and not go out casually. I suspect that there are many agents of Yingguo in the country, just waiting for Mr. Wei to go out and kidnap and assassinate or something."

"You've watched too many movies. How can there be so many fights in this world?"

"Hehe, if you say something like this, you're either stupid or bad, or it's a stupid and bad half a million."

"Wow, thinking about Mr. Wei being kidnapped by secret agents as soon as he left the country, he was locked in an underground dark room and forced to research and develop the elixir of life every day. It's really terrifying."

When Wei Kang saw these comments on the Internet, he couldn't help laughing and crying, thinking that netizens really have big brains.

But the worries of the netizens were in vain, because he was just a scientific research nerd, except for his family and the company, and the occasional appointment with Dr. Jian for dinner, he basically didn't go anywhere.

He has already made arrangements. After the new headquarters is built, he will move his family directly to the company and live in the villa in the back mountain. If it is not necessary, he will never go out again.

This time, Gu Xian and Sheng Changya will receive the Nobel Prize on their behalf, and the award ceremony will be broadcast live on the official website at midnight that night.

Wei Kang was at home at the time, and when the time came, Anmei reminded him to watch the live broadcast.

Turn on the TV, and the scene of the award ceremony has been automatically projected onto the 100-inch large screen.

In the magnificent city hall, scientists from all over the world gathered together to participate in the grand event.

The camera quickly showed Gu Xian. He was wearing a black dress, and he looked a little nervous, but it didn't affect his performance.

After receiving the award, he proficiently delivered a speech in English to express his gratitude on behalf of Wei Kang.

The whole process was smooth and flawless, and it is conceivable that he must have done many rehearsals in private.

At the subsequent dinner party, many shots focused on the table between Gu Xian and Sheng Changyan, and the two looked excited and changed their hair.

Sitting on the sofa, Wei Kang smiled gratifiedly with the corners of his mouth curled up.

At the same time, the R\u0026D building of the Sanqing headquarters.

It was early morning, but the floor of the biological laboratory was still brightly lit.

Suddenly, a burst of cheers sounded, spreading far away in the silent air.

Not long after, the lights went out one by one.

A figure appeared on the first floor and disappeared into the night.

Tang Que was at the end, took out his mobile phone, and sent a message to Cao Yang: "The genetic medicine is finished, hurry up in the morning."

His footsteps are a little sloppy, and he has to work overtime continuously, even if he is as strong as him, he can't bear it.

Soon, he opened the door of the dormitory, followed a familiar route, walked to the bed in the dark, and threw himself on the bed straight.

Almost instantly, he fell asleep.

Before losing consciousness, a thought flashed through his mind.

"It's too troublesome, I still have to develop a convenient and simple gene editing tool by myself!"