动物实验,帕金森氏病 z-bo 2020-09-14 332

　　The latest achievements of Shanghai scientists bring dawn to the treatment of Parkinson and other nerve damage and degenerative diseases

　　Once it takes a lifetime, even if some parts are worn out, there is almost no possibility of replacement. This is the human nervous system. This characteristic has brought endless troubles to humans: some functional damages cause blindness and paralysis, some degenerative changes cause Parkinson’s disease, Alzheimer’s disease... Recently, Shanghai scientists have used the latest gene editing technology to unearth The potential of nerve cells to become "super substitutes" has brought new light to the treatment of nerve damage and neurodegenerative diseases. Late last night, the international authoritative academic journal "Cell" published online the results of Shanghai scientists.

　　This time, it is a type of nerve cells called glial cells that have been given the potential of "super replacement" by scientists. There are a large number of these cells, and their main job is to provide nutrients for neurons, which is quite "free". "Why not extract a part of glial cells from the patient's body and transform them into neuronal cells that can take on more important tasks?" Five years ago, the idea was born by Yang Hui, a researcher at the Center for Excellence in Brain Science and Intelligent Technology of the Chinese Academy of Sciences.

　　In real life, it is too difficult to find a "substitute" for damaged or prematurely degenerated neurons: the use of embryonic stem cells has ethical problems, the use of induced pluripotent stem cells is too easy to induce tumors, and the direct transplantation of nerve cells is easy to produce rejection and Survival and integration will also be problematic... "If the idea of finding a'substitute' can be realized, it will benefit tens of millions of patients." Yang Hui, for example, said that many eye diseases, such as glaucoma and ischemic retinopathy, will cause optic ganglion cells. Death, which can lead to permanent blindness. According to statistics, the number of blind people from glaucoma alone exceeds 10 million in the world.

　　This is also a fiercely competitive field in the world. Yang Hui's research group, who is good at gene editing technology, has explored a unique path: they have discovered a small RNA editing tool from foreign scientific research reports that can enter organisms safely to carry out genes. Edit, so that glial cells are transformed into pluripotent stem cells, and then re-differentiated into neuronal cells that scientists need.

　　According to Zhou Haibo, the co-corresponding author of the paper and a postdoctoral fellow from the Center for Excellence in Brain Intelligence of the Chinese Academy of Sciences, they first designed a specific marker Muller glial cell and its expression CasRx system (a gene editing system) in cells in vitro, and then "packaged all the components "Injected under the retina of mice with permanent visual impairment due to damage to optic ganglion cells. About a month later, they were pleasantly surprised to find that the blind mice had light perception again-which means that the optic ganglion cells transdifferentiated from Muller glial cells can produce corresponding signals to light stimulation like normal cells, and It also establishes a functional connection with the correct brain area in the brain through the optic nerve, and transmits visual signals to the brain.

　　In a mouse model of Parkinson's disease, this technique has also been successful. Parkinson's disease is mainly caused by the loss of dopamine neurons in the substantia nigra of the brain. This time, the researchers took a fancy to the astrocytes in the striatum in the brain region of the substantia nigra, and transdifferentiated some of them into dopamine neuron cells, successfully making up for the lost dopamine neuron function in the substantia nigra— -The exercise ability of Parkinson's disease mice has been significantly improved.

　　"Cell" magazine reviewers believe that this research "gives an elegant and exciting case" "shows a new perspective, and may be widely used." Pu Muming, academician of the Chinese Academy of Sciences and academic director of the Center for Excellence in Brain Intelligence of the Chinese Academy of Sciences, said that he will advance the research into non-human primate experiments as soon as possible. "If significant results are also achieved, people will see more hope for clinical applications."