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, ...

Both symptomatic and asymptomatic individuals with pathogenic TTR variants had higher serum GFAP levels than healthy controls. Glial fibrillary acidic protein (GFAP) is a potential biomarker for early ...

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 ...

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 ...

Here, we demonstrate the use of AAVs for efficient gene transfer within the axolotl brain, the spinal cord, and the retina. We show that serotypes AAV8, AAV9, and AAVPHP.eB are suitable viral vectors ...

Abstract: The detection of Glial Fibrillary Acidic Protein (GFAP) is a crucial biomarker for diagnosing neurological conditions such as traumatic brain injuries (TBI) and astrocytoma. This paper ...

About AAVB-081 program AAVB-081 is an intra-retinal AAV8-based dual hybrid product targeting MYO7A-associated Usher syndrome (USH1B). AAVantgarde’s dual hybrid platform uses two AAV8 vectors, each ...

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 ...

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 ...