Bardet-Biedl syndrome (BBS) is a group of rare autosomal recessive ciliopathies characterized by dysfunction of primary cilia, which affects multiple organ systems and leads to early-onset obesity, ...

Glial fibrillary acidic protein (GFAP) is a potential biomarker for early, preclinical changes in patients with transthyretin amyloid polyneuropathy (ATTR-PN) and could aid in early detection, ...

本研究系统比较了AAV8、AAVMYO和AAVMYO2三种腺相关病毒（AAV）对骨骼肌中单核细胞（包括内皮细胞、免疫细胞、肌肉干细胞MuSCs和纤维脂肪祖细胞FAPs）的转导效率差异。通过静脉（i.v.）和肌内（IM）注射携带tdTomato报告基因的病毒，结合流式细胞术和单分子荧光原位杂交（smFISH），揭示了AAV血清型 ...

Using the CAG promoter to drive expression of fluorescent proteins, we find that neurons rather than ependymoglia are preferentially labeled by AAV8, AAV9, and AAVPHP.eB. Finally, we demonstrate the ...

Mutations in the BBS10 gene are the second most common cause of Bardet-Biedl syndrome (BBS). Researchers from Meiragtx Ltd. aimed to optimize and identify an AAV vector carrying the human (h)BBS10 ...

Glioma is the most common primary brain tumor with its early detection remaining a challenge. Autoantibodies against glial fibrillary acidic protein (GFAP) have shown the highest differential ...

Following numerous rounds of screening in a variety of species, the investigators developed the AAV8-based vector GS1196, which expresses C1-INH, for the treatment of individuals with HAE. After a ...

As part of it, GFAP and NfL levels in blood samples were measured at least once for 243 participants, of whom 108 were diagnosed with SPMS and 135 with PPMS.. GFAP provides structure to astrocytes ...