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#基因疗法 ✕
B
[Chinese scientists “install switches” in the brain to treat epilepsy. International colleagues: Other gene therapies cannot achieve this.] On November 5, the New England Journal of Medicine (NEJM) published a breakthrough study completed by Chinese scientists—the world’s first human clinical study of chemical genetic neuromodulation. In the comments issued by international colleagues, they believe that this study innovatively combines gene therapy with small molecule drugs, allowing the intensity of treatment to be adjusted as needed, which is something that cannot be achieved with other existing types of gene therapy. The research was jointly completed by Professor Zhao Guoguang’s team from Xuanwu Hospital of Capital Medical University, Professor Luo Minmin’s team from the Beijing Institute of Brain Science and Brain Integration, and Kinda Jiuzhou (Beijing) Biotechnology Co., Ltd. Its biggest innovation is that through a single gene delivery, the neurons in a specific brain area express an "artificially designed receptor" that does not exist in nature. It only recognizes a specific small molecule drug. It is "silent" when not taking the drug and is activated when taking the drug.
#gene therapy
#brain science
#Chemical
#medicine
#molecular
#Gene
#Futures Market Intelligence
#TMT Industry Observation
11-05 21:49
B
On November 2, UniQure’s U.S. stocks rose 12% before the market opened after the FDA accepted priority review for UniQure’s Huntington’s disease gene therapy application.
#gene therapy
#Gene
#U.S. stock trends
#medicine
#Pharmaceutical business
11-02 20:13
B
["Prime assembly" can accurately stitch long DNA fragments] On September 21, a research team from Boston Children's Hospital in the United States published a new genome editing method called "prime assembly" in the latest issue of "Nature" magazine. This technology can precisely "stitch" long DNA fragments to precise programmable sites in the genome of living cells, and repair multiple mutations simultaneously in one operation. This advance could lead to the development of universal gene therapies suitable for most patients.
#gene therapy
#DNA
#genome
#programming
#Gene
#cell
#Global market intelligence
09-21 06:37
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