AAV-Mediated Gene Transfer of WDR45 Corrects Neurological Deficits in the Mouse Model of Beta-Propeller Protein-Associated Neurodegeneration.

• Tác giả: Martha Bishop, Maria Carla Carisi, Uma Chandrachud, Yulia Grishchuk, Brandon Meyerink, Louis Jean Pilaz, Madison Sangster, Claire Shamber
• Ngôn ngữ: eng
• Ký hiệu phân loại: 932.013 Middle Kingdom and second intermediate periods, 2052—1570 B.C.
• Thông tin xuất bản: United States : Human gene therapy , 2025
• Bộ sưu tập: NCBI
• ID: 224024

Tóm tắt

Beta-propeller protein-associated neurodegeneration (BPAN) is an ultra-rare, X-linked dominant, neurodevelopmental, and neurodegenerative disease caused by loss-of-function mutations in the