Gene and cell therapies for Parkinson’s make headway

IF 41.7 1区 生物学 Q1 BIOTECHNOLOGY & APPLIED MICROBIOLOGY Nature biotechnology Pub Date : 2025-02-14 DOI:10.1038/s41587-025-02581-4
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帕金森病基因和细胞疗法取得进展
1月份,一项治疗帕金森病的实验性基因疗法进入了第二阶段研究。拜耳(bayer)旗下的AskBio公司将在中度帕金森氏症成人患者中进行随机双盲研究,将一种腺相关病毒血清型2 (AAV)送入大脑。AB-1005疗法将把人类神经胶质细胞系衍生的神经营养因子(GDNF)基因注入神经元,以促进壳核(受帕金森病影响的区域)的再生。1期结果显示AB-1005耐受性良好,无严重不良事件。拜耳的另一家公司BlueRock Therapeutics宣布,将在2025年上半年启动一项双盲三期试验,研究移植的产生多巴胺的细胞是否能阻止黑质退化并恢复运动功能。这种疗法是一种异体多能干细胞衍生疗法。在一期研究中,该药物在术后24个月无严重不良反应。
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来源期刊
Nature biotechnology
Nature biotechnology 工程技术-生物工程与应用微生物
CiteScore
63.00
自引率
1.70%
发文量
382
审稿时长
3 months
期刊介绍: Nature Biotechnology is a monthly journal that focuses on the science and business of biotechnology. It covers a wide range of topics including technology/methodology advancements in the biological, biomedical, agricultural, and environmental sciences. The journal also explores the commercial, political, ethical, legal, and societal aspects of this research. The journal serves researchers by providing peer-reviewed research papers in the field of biotechnology. It also serves the business community by delivering news about research developments. This approach ensures that both the scientific and business communities are well-informed and able to stay up-to-date on the latest advancements and opportunities in the field. Some key areas of interest in which the journal actively seeks research papers include molecular engineering of nucleic acids and proteins, molecular therapy, large-scale biology, computational biology, regenerative medicine, imaging technology, analytical biotechnology, applied immunology, food and agricultural biotechnology, and environmental biotechnology. In summary, Nature Biotechnology is a comprehensive journal that covers both the scientific and business aspects of biotechnology. It strives to provide researchers with valuable research papers and news while also delivering important scientific advancements to the business community.
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