FRATAXIN gene therapy

• Main Authors: CHANG, JING, CHUNG, SEN DIPANJAN, GRAY JOHN T
• Format: Patent
• Language: Chinese; English
• Published: 04.06.2024
• 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

The present invention provides compositions and methods for stimulating the expression of the human fraxin gene. The compositions described herein can be used, for example, to produce gene and RNA equivalents optimized for expression in particular cell types. The compositions and methods can be used to treat Friedel's ataxia. Using the compositions and methods of the present disclosure, a plasmid (e.g., a viral vector) containing a human fraxin gene (hFXN) or an RNA equivalent thereof can be administered to a patient suffering from Freedehi ataxia (e.g., a mammalian patient, such as a human patient). 本发明提供用于刺激人frataxin基因的表达的组合物和方法。本文所述的组合物能用于例如产生针对特定细胞类型中的表达而优化的基因和RNA等效物。所述组合物和方法能用于治疗弗里德赖希共济失调。使用本公开的组合物和方法，可以向患有弗里德赖希共济失调的患者(例如哺乳动物患者，诸如人类患者)施用含有人frataxin基因(hFXN)或它的RNA等效物的质粒(例如，病毒载体)。