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Alzheimer's disease (AD) is the most common neurodegenerative disease among the elderly.
Among them, familial Alzheimer's disease (FAD) is a genetic disease caused by genetic mutations
.
These patients have mutations in genes related to amyloid processing, leading to AD symptoms at a younger age
Recently, an international research team led by scientists from the Hong Kong University of Science and Technology has developed an innovative technology that enables extensive gene editing in the brain
.
In the AD mouse model, the pathogenesis of AD was significantly reduced
In this study, scientists designed a gene editing therapy based on CRISPR-Cas9
.
This gene editing therapy, after being injected into the brain, can specifically disrupt the amyloid precursor (APP) gene carrying genetic mutations in a wide range of brain tissues, thereby significantly reducing the level of amyloid protein in the animal's brain
▲The deposition of amyloid in the brain area of mice treated with gene editing therapy (right) was significantly reduced (picture source: reference [1])
Moreover, the researchers also designed an AAV vector that can cross the blood-brain barrier, which can effectively deliver the gene editing system to the brain with only one intravenous injection
.
Experimental results show that this therapy can also alleviate the deposition of amyloid, and alleviate the proliferation of microglia, neurodegeneration, and cognitive impairment
Reference materials:
[1] HKUST Scientists Develop Genome-editing Strategy for Potential Alzheimer's Disease Therapy.
[2] Duan et al.
(The original text has been deleted)
