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▎Editor of WuXi AppTec's content team In 1907, German psychiatrist Alois Alzheimer (Alois Alzheimer) discovered during an investigation that a female mentally ill patient suffered from memory loss and mental confusion
.
He then recorded this particular illness and wrote it on the case report
.
In the following decades, scientists have gradually learned more about this pattern of disease, and have also given this disease a name, namely Alzheimer's disease
.
At present, the main consideration in the research and development of Alzheimer's disease treatment is to reduce those abnormal proteins that damage neurons, that is, beta amyloid, to restore nerve function
.
In addition to using drugs to control disease progression, in fact, some patients with Alzheimer's disease can benefit from emerging gene editing technologies
.
Familial Alzheimer's disease patients usually get pathogenic genetic factors from their parents, and these patients can be diagnosed even before symptoms appear
.
Unfortunately, there is currently no effective means to prevent the disease from happening.
They can only wait for Alzheimer's disease to come
.
In China alone, there are more than 500,000 such patients
.
However, a study in "Nature Biomedical Engineering" brought good news
.
The team from the Hong Kong University of Science and Technology has designed tools that can modify pathogenic mutations based on gene editing technology, and has achieved noteworthy results in mouse models
.
▲Leader of this research, Professor Nancy Ip (second from right) and team members (photo source: Hong Kong University of Science and Technology; Credit: Hong Kong University of Science and Technology) In the past, gene editing tools wanted to enter the brain non-invasively to correct mutations , The biggest obstacle is the blood-brain barrier around the brain
.
Originally, this barrier is like a city wall, protecting the brain from toxins and pathogens entering the brain, but it will also indiscriminately block drugs or therapeutic tools
.
The new system developed by the research team is based on an adenovirus vector, which carries two key genes: Cas9 and a guide RNA for the target pathogenic mutation, which can be used to knock out the pathogenicity of the β-amyloid precursor protein coding gene.
Mutation
.
In the test, the researchers delivered gene editing tools into diseased mice through hippocampal injection and intravenous injection
.
The results found that hippocampal injection can significantly reduce the level of amyloid β in disease model mice.
After 6 months of gene editing treatment, the CA area of the hippocampus and the area containing abnormal β amyloid in the hippocampus decreased respectively.
Which was 72.
9% and 77.
9%
.
In addition, these tools can be delivered to the entire brain, which means that they will not only work in some brain regions, but will help brain cells to improve the negative effects of disease-causing mutations in all aspects
.
▲By injecting editing tools into the hippocampus, the level of amyloid β in different brain regions can be reduced
.
(Picture source: Reference [2]; Credit: Hong Kong University of Science and Technology) After the researcher has slightly improved the carrier, the same effect can be obtained by intravenous injection
.
Various health indicators of the brain of disease model mice are gradually returning to normal.
For example, the reduction of gliosis and the restoration of nerve function mean that the brain of mice is gradually becoming healthy, and gene editing tools can cross the blood-brain barrier and take effect
.
▲In mice treated by intravenous injection, the level of amyloid β in the brain was significantly lower than that in the control group (picture source: reference [2]; Credit: Hong Kong University of Science and Technology).
In addition, this effect is not only temporary, as long as After one injection of the gene editing vector, even after 6 months of treatment, the level of amyloid β in the brains of disease model mice is still maintained at a very low level, which is equivalent to 1/3 of their lifespan
.
Overall, the researchers did not observe any side effects in the treated mice
.
"This is the first gene editing tool that can work across the entire brain and reduce the symptoms of Alzheimer's disease.
It is indeed an exciting discovery," said Professor Ye Yuru from the Hong Kong University of Science and Technology, who is also the head of the research.
"For the treatment of hereditary brain diseases, our research can be regarded as a landmark discovery
.
In the future, it will also help to develop precise medical methods for the treatment of hereditary neurodegenerative diseases
.
References ：[1] HKUST scientists develop genome-editing strategy for potential Alzheimer's disease therapy, Retrieved August 17th, from https:// Duan, Y.
, Ye, T.
, Qu, Z.
et al.
Brain-wide Cas9-mediated cleavage of a gene causing familial Alzheimer's disease alleviates amyloid-related pathologies in mice.
Nat Biomed Eng (2021).
https://doi.
org/10.
1038/s41551-021- 00759-0
.
He then recorded this particular illness and wrote it on the case report
.
In the following decades, scientists have gradually learned more about this pattern of disease, and have also given this disease a name, namely Alzheimer's disease
.
At present, the main consideration in the research and development of Alzheimer's disease treatment is to reduce those abnormal proteins that damage neurons, that is, beta amyloid, to restore nerve function
.
In addition to using drugs to control disease progression, in fact, some patients with Alzheimer's disease can benefit from emerging gene editing technologies
.
Familial Alzheimer's disease patients usually get pathogenic genetic factors from their parents, and these patients can be diagnosed even before symptoms appear
.
Unfortunately, there is currently no effective means to prevent the disease from happening.
They can only wait for Alzheimer's disease to come
.
In China alone, there are more than 500,000 such patients
.
However, a study in "Nature Biomedical Engineering" brought good news
.
The team from the Hong Kong University of Science and Technology has designed tools that can modify pathogenic mutations based on gene editing technology, and has achieved noteworthy results in mouse models
.
▲Leader of this research, Professor Nancy Ip (second from right) and team members (photo source: Hong Kong University of Science and Technology; Credit: Hong Kong University of Science and Technology) In the past, gene editing tools wanted to enter the brain non-invasively to correct mutations , The biggest obstacle is the blood-brain barrier around the brain
.
Originally, this barrier is like a city wall, protecting the brain from toxins and pathogens entering the brain, but it will also indiscriminately block drugs or therapeutic tools
.
The new system developed by the research team is based on an adenovirus vector, which carries two key genes: Cas9 and a guide RNA for the target pathogenic mutation, which can be used to knock out the pathogenicity of the β-amyloid precursor protein coding gene.
Mutation
.
In the test, the researchers delivered gene editing tools into diseased mice through hippocampal injection and intravenous injection
.
The results found that hippocampal injection can significantly reduce the level of amyloid β in disease model mice.
After 6 months of gene editing treatment, the CA area of the hippocampus and the area containing abnormal β amyloid in the hippocampus decreased respectively.
Which was 72.
9% and 77.
9%
.
In addition, these tools can be delivered to the entire brain, which means that they will not only work in some brain regions, but will help brain cells to improve the negative effects of disease-causing mutations in all aspects
.
▲By injecting editing tools into the hippocampus, the level of amyloid β in different brain regions can be reduced
.
(Picture source: Reference [2]; Credit: Hong Kong University of Science and Technology) After the researcher has slightly improved the carrier, the same effect can be obtained by intravenous injection
.
Various health indicators of the brain of disease model mice are gradually returning to normal.
For example, the reduction of gliosis and the restoration of nerve function mean that the brain of mice is gradually becoming healthy, and gene editing tools can cross the blood-brain barrier and take effect
.
▲In mice treated by intravenous injection, the level of amyloid β in the brain was significantly lower than that in the control group (picture source: reference [2]; Credit: Hong Kong University of Science and Technology).
In addition, this effect is not only temporary, as long as After one injection of the gene editing vector, even after 6 months of treatment, the level of amyloid β in the brains of disease model mice is still maintained at a very low level, which is equivalent to 1/3 of their lifespan
.
Overall, the researchers did not observe any side effects in the treated mice
.
"This is the first gene editing tool that can work across the entire brain and reduce the symptoms of Alzheimer's disease.
It is indeed an exciting discovery," said Professor Ye Yuru from the Hong Kong University of Science and Technology, who is also the head of the research.
"For the treatment of hereditary brain diseases, our research can be regarded as a landmark discovery
.
In the future, it will also help to develop precise medical methods for the treatment of hereditary neurodegenerative diseases
.
References ：[1] HKUST scientists develop genome-editing strategy for potential Alzheimer's disease therapy, Retrieved August 17th, from https:// Duan, Y.
, Ye, T.
, Qu, Z.
et al.
Brain-wide Cas9-mediated cleavage of a gene causing familial Alzheimer's disease alleviates amyloid-related pathologies in mice.
Nat Biomed Eng (2021).
https://doi.
org/10.
1038/s41551-021- 00759-0
