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

The two parties reached a consensus to use gene editing tools to completely knock out the extra A bases on the atp7a gene to fundamentally treat menkes patients.

However, there are different opinions when it comes to which gene editing tool to choose.

After entering the 21st century, with the development of biotechnology, scientists have continued to develop to achieve gene editing. So far, three generations of new technologies have been produced, namely zfn (zinc finger nuclease) and talens (transcription activator-like effector).

Three gene editing tools: nuclease) and CRISPR/Cas9.

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

It uses zinc finger proteins that are widely found in eukaryotes (from yeast to humans) to recognize and bind a specific triplet base, thereby achieving the purpose of site-specific shearing of DNA.

talen is a second-generation gene editing tool that appeared ten years ago. It has achieved considerable technological breakthroughs and can complete gene editing work in a more efficient manner, and rarely accidentally cuts off non-target DNA sequences. So far,

, received high praise.

Talens uses a natural protein secreted by plant bacteria to recognize specific DNA base pairs, binds to the target DNA and cuts the DNA chain at a specific site, thereby introducing new genetic material.

Compared with zfn, talen can target longer gene sequences and is easier to construct.

But until now, there has been no low-cost and publicly available method to quickly generate large numbers of tales.

crispr-cas 9 is the third generation and latest gene editing tool. The inventors are two female scientists who also won the Nobel Prize last year.

This is a groundbreaking new technology that is fundamentally different from the first and second generation genome editing technologies, so it is often called the "magic scissor". It is precisely because of its emergence that genome editing technology has gained

gained global popularity.

CRISPR was originally a natural immune system invented by bacteria during the evolution process billions of years ago. After being invaded by a virus, some bacteria can store a small fragment of the viral gene into a storage space called CRISPR in their own DNA.

When the virus invades again, once the bacteria find the same part as the stored fragment, the Cas9 protein will deform, accurately jam the viral DNA and wield the "scissors" without hesitation.

CRISPR can produce changes or mutations in the genome more efficiently than other gene editing technologies such as Talen.

It uses guide RNA to find the DNA that needs to be cut as a target, and then uses Cas9, an enzyme specifically designed to cut DNA, to operate, thereby accurately cutting the DNA target and achieving 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 operate genes in an extremely simple way.

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

Although the most popular gene editing technology now is crisp-cas9, the three editing systems actually have their own advantages and disadvantages.

It can only be said that each researcher needs to choose the most appropriate tool based on his or her own topic.

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 single-gene genetic diseases and can be treated by modifying genes.

As for more common diseases, including cancer, hypertension and other chronic diseases, it is currently impossible to treat even dozens of genes, because their causative factors are very complex.

Tang Que was cautious and did not want to use the latest crisp-cas9 tool.

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

Secondly, its underlying patent belongs to Yingguo Company. If it is to be used commercially, it involves 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 are no problems in this regard.

There is no problem if Cao Yang uses crisp-cas9 for personal development. The patentee does not restrict its use in scientific research, and his long-term research and efforts are also directed at this tool.

However, as a commercial pharmaceutical company, Sanqing has more aspects to consider and is more inclined to choose tools without patent problems.

However, the problem was not big. After friendly negotiations, the two parties decided to use the zfn gene editing tool to knock out the gene.

Because of the interaction of DNA-binding elements, the accuracy of the ZFN method is unpredictable, so proteins that map to each DNA sequence need to be constructed and tested.

This undoubtedly requires a high degree of expertise and is also 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, seven or eight more menkes patients were brought to Kunming by their families, where they underwent detailed testing on Sanqing's gene sequencer to find the gene targets and carry out targeted preliminary preparations.

The entire laboratory is like a machine running at full speed for the same goal.

Gu Zhi

******

Time passed quickly, and Wei Kang was waiting for the Nobel Prize Award 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 pick it up on his behalf.

This news instantly sparked a heated discussion.

Netizens expressed their understanding one after another.

"General Wei is now a Chinese national soldier. It is a strategic material. He must not go abroad, otherwise he will definitely be detained by the shameless Eagle Kingdom."

"Yes, Yingguo is so despicable and shameless. I strongly recommend 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 to kidnap and assassinate."

"You've watched too many movies, right? There's so much fighting and killing in this world."

"Haha, if you say something like this, you are either stupid or bad, or else you are both stupid and bad."

"Wow, it's really scary to think that as soon as Director General Wei left the country, he was kidnapped by agents, locked up in a small underground dark room, and forced to develop the elixir of life every day."

When Wei Kang saw these comments on the Internet, he couldn't help but laugh and cry, thinking that netizens are really imaginative.

However, netizens' worries were in vain, because he is just a scientific research geek. He basically doesn't go anywhere except home and work every day, as well as the occasional date with Dr. Jian for dinner.

He has already arranged that after the new headquarters is built, he will move his family directly to the company and live in a villa in the back mountain. He will never leave home again unless necessary.

The recipients of the Nobel Prize this time are Gu Xian and Sheng Changya, and the award ceremony will be broadcast live on the official website at 0:00 that night.

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

When you turn on the TV, the image 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 looked a little nervous, but this did not affect his performance.

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

The whole process was smooth and impeccable. It is conceivable that he must have rehearsed it many times in private.

At the dinner that followed, many times the camera focused on the table of Gu Xian and Sheng Changya, both of whom looked excited and had their hair changed.

Wei Kang sat on the sofa, with the corners of his mouth raised, smiling happily.

At the same time, the R&D building of Sanqing headquarters.

It was already early in the morning, but the biology laboratory floor was still brightly lit.

Suddenly, a burst of cheers sounded.

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

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

Tang Que walked at the end. He took out his mobile phone and sent a message to Cao Yang: "The genetic medicine is ready. Come quickly in the morning."

His steps were a little sloppy and he worked overtime continuously, which was too much even for him as strong as he was.

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

Almost instantly, he fell into sleep.

Before losing consciousness, a thought flashed through his mind.
Chapter completed!