用于神经变性疾病的基因疗法

• Format: Patent
• Language: Chinese
• Published: 31.12.2024
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; ENZYMOLOGY; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPIRITS; VINEGAR; WINE

本公开在一些方面涉及用于治疗神经变性疾病(例如肌萎缩性脊髓侧索硬化症(ALS)和/或额颞叶痴呆(FTD)、阿兹海默氏病、高雪氏病、帕金森氏病、路易体痴呆或溶酶体贮积病)的组合物和方法。在一些实施方案中，本公开提供了表达构建体，所述表达构建体包含编码一个或多个抑制性核酸(例如靶向C9orf72、TMEM106B、ATNX2、RPS25等)、野生型C9orf72蛋白或其部分或上述物质的任何组合的转基因。在一些实施方案中，本公开提供了通过将这些表达构建体施用到需要的对象来治疗ALS/FTD的方法。 The disclosure relates, in some aspects, to compositions and methods for treatment of neurodegenerative diseases (e.g., amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD), Alzheimer's disease, Gaucher disease, Parkinson's disease, Lewy body dementia, or a lysosomal storage disease). In some embodiments, the disclosure provides expression constructs comprising a transgene encoding one or more inhibitory nucleic acids (e.g., targeting C9orf72, TMEM106B, ATNX2, RPS25, etc.), wild-type C9orf72 protein or a portion thereof, or any combination of the foregoing. In some embodiments, the disclosure provides methods of ALS/FTD by administering such expression constructs to a subject in need thereof.