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In 1907, German psychiatrist Alois Alzheimer (Alois Alzheimer) found in an investigation that a female mentally ill patient suffered from memory loss and mental confusion
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
In addition to using drugs to control disease progression, in fact, some patients with Alzheimer's disease can benefit from emerging gene editing technologies
Unfortunately, there is currently no effective means to prevent the disease from happening.
However, a study in "Nature Biomedical Engineering" brought good news
In the past, when gene editing tools wanted to enter the brain to correct mutations non-invasively, the biggest obstacle was the blood-brain barrier around the brain
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.
The results found that hippocampus injection can significantly reduce the level of amyloid β in disease model mice.
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 levels of β-amyloid in different brain regions can be reduced
After the researchers slightly improved the carrier, the same effect can be obtained by intravenous injection
▲The level of beta amyloid in the brain of mice treated by intravenous injection was significantly lower than that of the control group (picture source: reference [2]; Credit: Hong Kong University of Science and Technology)
In addition, this effect is not only a short-term appearance.
Note: The original text has been deleted
Reference materials:
[1] HKUST scientists develop genome-editing strategy for potential Alzheimer's disease therapy, Retrieved August 17th, from https:// Duan, Y.
