This doctor has a system
Chapter 541 604 Searching for God
Chapter 541 604. Searching for God
Gene therapy sounds high-sounding, but in essence it means introducing a DNA sequence containing a normal gene into cells via a vector, causing them to produce the product of the target gene, thereby achieving the purpose of treating the disease caused by the gene defect.
Professor Lasse West designed an ingenious treatment plan, choosing recombinant adeno-associated virus type 9 as the vector to deliver a functional copy of the gene to the central nervous system via intrathecal administration. This route of administration has also been widely used in preclinical and clinical studies of the treatment of central nervous system diseases.
Theoretically, SPG50 is an ideal candidate for gene therapy: the AP4M1 gene coding sequence is very short and very suitable for self-complementary adeno-associated virus vectors.
AAV9/AP4M1构建体可大量表达 AP4M1蛋白,从而帮助患者恢复正常的 AP4M1蛋白功能。
What is exciting is that in the preclinical research stage, intrathecal AAV9/AP4M1 gene therapy showed safety and effectiveness, which made the participants and Parton's family overjoyed.
In order to obtain the best therapeutic effect through early intervention and higher dose, the target dose was set at 5 ml and 1.5×10^15 vector genomes.
Based on the preclinical research provided by the treatment team, the local Ministry of Health officially approved the Phase I clinical trial of the treatment plan; in April last year, the clinical trial was approved by the institutional ethics committee. In June of the same year, after two months of preparation, this clinical trial with only one person was officially launched.
But the results were not satisfactory.
But in fact, it was not without results. First, the researchers injected AAV9-AP4M1 into Patton's body through lumbar puncture intrathecal injection, and also carried out AP4M1 protein immunosuppressive treatment.
Patton did not experience any obvious side effects, which shows that the trial was safe and tolerable, but the efficacy evaluation was far from the expected results.
To sum it up in one sentence: safe and useless.
Everyone was very disappointed, which meant that their efforts during this period might be useless.
Professor Lasse West carefully reviewed his experimental process in the hope of finding possible flaws, but he failed.
The unwilling crowd conducted animal experiments again, only to find that the effect of the drug on mice was just as good as before. After intrathecal injection, the mice quickly caught up with normal mice in development.
"Fuck!" Professor Lasse West cursed.
"Professor, we need some patience," the assistant said. "After all, mice are different from humans."
Professor Lasse West certainly knew that the physiological structure of mice was different from that of humans, and in fact he would definitely be able to find out this difference in the end.
But the problem is that patients can't wait. Doctors have evaluated that if no drug for this disease is developed within one year, Patton's great-grandmother will come to take him away.
This meant that everything they had done was in vain. Since the customs and habits in the United States were different from those in China, the participants could not even have a meal.
Not even being able to have a meal sounds miserable.
After careful consideration, Professor Lasse West decided to write an article to calm down. He wrote down all the extremely detailed data of the clinical trial on one person. At the end of the article, he wrote: I hope God can appear and save this poor child, if there really is a God in this world.
Jones, the editor of Natural Medicine, saw the article first and felt a headache.
He did not want to accept the article, even though its author was the prestigious professor of neurology, Lasse West.
"It's too long. This will take up at least 5 pages."
Jones asked Professor Lasse West to make changes to the content of the article, and the latter readily agreed. Then he resent the manuscript two days later, which took up four pages.
"You can't do this!"
"There's no way to shorten it any further," Professor Lasse West argued. "I have to write down all the necessary steps."
Clinical trials are a very complex process. Can 1 to 2 words describe it clearly?
For general readers, it is OK, because they don't understand the details and there is no point in reading it. But for scholars whose research direction is in this area, the more detailed the data listed in the article, the better.
This is the same as steaming buns. If you don’t write down the specific steps clearly, no one will know what filling you are using in the buns.
Because of Professor Lasse West's special status and his good personal relationship, Editor Jones was in a dilemma.
"Well, try voting naturally."
"Fuck you!" Professor Lasse West cursed directly, "Your journals don't accept such stuff, so naturally they will accept it?"
Jones said he couldn't accept this.
First: I don’t say hello. Sorry, I went to the wrong set.
First: Your article has a very high purpose. It is a clinical trial conducted on one person. It makes people feel grand and noble. It is a bit low-class to submit such a grand article to Nature Medicine. Nature, Cell, and New England Journal are the ideal places for it.
Second: We have very little space. What will you do to other authors? They work very hard! I have to throw away dozens of manuscripts every day. This is very unfair to them.
Third: It is a ridiculous method to look for God through an article. As we all know, God is a girl. I suggest you seek help from the Women and Children's Association, which is more reliable.
"Just tell me whether you will send it or not?" Professor Lasse West didn't want to listen to his nagging.
"It's not up to me to decide. It needs to be approved by independent reviewers." Jones began to practice Tai Chi.
Professor Lasse West knew what he was thinking, but there was nothing he could do about it. He also knew that this was a case of a strongman locking the man up.
Or maybe he wasn't strong enough, so he couldn't lock down the old man Jones.
"Then as soon as possible."
"Okay." Jones felt relieved when he saw that the other party no longer insisted. "I will help you urge the reviewers to give their suggestions as soon as possible." He said comfortingly.
Professor Lasse West chuckled after hanging up the phone. His old friend must have thought he was going to give up, but he didn't.
After a while, the phone rang again.
"Hey, this is against the rules." A helpless voice came over, "Can't you just publish in another journal? I can help you."
“Do other journals have the same impact as Nature Medicine?”
"I saw from the backstage that the article was sent to Browning Rudolph of Cambridge University Medical School; Gallop Willey of Paris Saclay-Perpignan Medical School..."
"And Feng Gao from ZZ University, Professor Gao Feng, is this year's Lasker Award winner for medical science."
Okay, a British professor, a French professor, and finally a Chinese professor, very good!
Professor Lasse West was very angry. Damn Jones, it was obvious that the old guy was guarding against him.
He did know Professor Rudolph Browning of the Cambridge University Medical School, but he was very arrogant and difficult to deal with.
Once when he was attending a meeting, he sat next to the other person and wanted to say hello in a friendly manner, but Browning Rudolph was very indifferent. No matter what he said, the other person just nodded, said oh, um, or ah.
Gallup Willey of the Paris-Saclay-Perpignan Medical School is completely unfamiliar to the Chinese.
However, I heard that Professor Gao Feng has successful experience in gene therapy. I wonder if he can find anything from the article.
The weather in Cambridge is always gray, and it rained today, so there were even fewer people on the road. "You can't go anywhere in this weather," Professor Browning Rudolph complained, "No wonder everyone likes to be gay."
The assistant's CPU was almost smoking. "Professor, they can date the opposite sex at home. This is not an excuse to be gay."
"You know so much."
When you fall in love with the opposite sex, you must do it in sunny weather so that the spark of love can be ignited. If it can be ignited, then do it outdoors.
"Really, that's how it was when I was young," said Professor Browning Rudolph.
"But now there are more and more gays, and they don't even hide from people anymore!"
“I don’t understand it either, but the law says it’s their right,” the assistant laughed.
"Bullshit rights!" said Professor Browning Rudolph with disgust.
"Let's not talk about this anymore." The assistant ended the topic in time, "There is an article that needs your review, and the editor is pressing for approval."
Professor Browning Rudolph didn't want to work, but after thinking about it, he sighed helplessly and said, "Bring it here."
When he first saw the article, he asked, "Why is it so long? What's wrong with the editor?"
You should know that natural medicine has clear requirements on the word count for manuscripts. Professor Browning Rudolph knew at a glance that this manuscript did not meet the requirements.
It can be sent in this state, is there something special about it?
He put on his glasses and began to look at it carefully. Clinical trial? Or one person's?
Very good, you have successfully attracted the attention of the great straight professor.
Professor Browning Rudolph spent three days reading the entire manuscript. By this time, the weather in Cambridge had improved, and young men and women appeared on the streets, injecting new vitality into the city.
"I spent so much money on one person, but the effect is not good."
“Isn’t this a waste of public resources?”
Professor Browning Rudolph was a pragmatist and a firm believer in the saying that winners take their place, and he felt a little contempt for the author of this article.
"Then will it be rejected?" asked the assistant.
"No, pass it." Unexpectedly, Professor Browning Rudolph said this.
"He wants to find God, so let him find it."
The assistant agreed and began to write the review comments. Professor Browning Rudolph stretched and walked out of the office.
The assistant vaguely heard him mumbling: You little piece of trash, you have no strength, you can't handle it yourself, and you still want to find God, you are dreaming
It should be a new type of rap singing style, the assistant thought. As far as he knew, the professor was an amateur rapper who had been practicing for 2 and a half years.
Professor Gallop Willey of the Paris-Saclay-Perpignan Medical School exclaimed in amazement when he first saw the article.
"Wow, that's so cool!"
"They're actually conducting a clinical trial just for one patient! How romantic!"
That's how the French are, romance is in their blood.
Professor Gallup Willy is a typical French romantic. He inherited the good qualities of his ancestors, that is, he does everything quickly, and I hereby declare that this does not include surrender.
Two hours later he gave his review opinion: passed!
He also wrote one more sentence at the end: I hope God will show up soon.
God, no, Gao Feng is still reading the manuscript.
If this were to be published, it would take up at least four pages, he thought.
Moreover, there is little significance in publishing it. After all, gene therapy drugs need to be customized. The results of this experiment are not ideal, and others cannot get any reference.
However, it is not completely without merit. At least the designed experimental ideas are still very ingenious, which is why the animal experiments went smoothly.
So why didn't the intrathecal injection work on Patton?
Gao Feng yawned, which was obviously caused by the other party's lack of experience.
重组腺相关病毒载体 9型是良好的载体,它稳定高效,而且非常安全。AAV9/AP4M1构建体可大量表达 AP4M1蛋白,这两者在一起看着好像是天作之合。
But in fact, although mice are also warm-blooded animals, their normal body temperature is also around 36.5 degrees Celsius. Although they have certain similarities with humans at the genetic level, the genetic similarity between mice and humans is about 85%.
But in comparison, there are several special proteolytic enzymes in the human spinal canal that can destroy the stability of recombinant adeno-associated virus vector type 9, greatly reducing their effectiveness.
Thinking of this, Gao Feng was a little dissatisfied. Didn’t I mention this in my previous article published in Nature?
You can’t even copy homework?
He reread his article and felt a little embarrassed, as if he had wronged the other party. He had deleted this part before because of the length of the article.
"Fail!"
"Hey, man, there's nothing we can do. The reviewer rejected your article," said Jones, the editor of Nature Medicine, with a regretful tone. "Actually, I think it's pretty well written."
"roll!"
"But they all wrote their comments, I will forward them to you anonymously. No, that's not right!" Jones' tone became a little strange, "One reviewer seems to have written a lot of content."
"Oh my goodness! My friend!" he cried, "You may have actually met God!"
"What are you talking about?" Professor Lasse West asked curiously.
"It's the Chinese! The Chinese pointed out the problem at the end! Forget it, just see for yourself!" Editor Jones couldn't tell the truth from the false.
"Enzyme problem???" Professor Lasse West was stunned.
"Fuck!" he yelled. "Louis! Louis! Come here!"
Assistant Louis carefully read the review comments at the end of the article and fell into ecstasy. He was 90% sure that what the other party said was true.
"You are so wise and brave!" He said to the professor emotionally, "How did you come up with the idea of sending out the clinical trial process? This is really a genius idea."
If other people were present, they would certainly wonder, isn’t it common practice to summarize clinical trials and publish them as articles?
Where is his wisdom and bravery?
(End of this chapter)
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