DNL310 normalizes primary storage substrates and biomarkers of lysosomal dysfunction in neuronopathic MPS II: 2-year interim analysis of a phase 1/2 study

Akhil Bhalla, Khishig Tsogtbaatar,Ritesh Ravi,Meng Fang,Sonnet S. Davis,Imanol Zubizarreta,Ajith Karunakara,Yuda Zhu, Elizabeth Sinclair, Dominique Jacquemet-Engelhart, Shyeilla V. Dhuria,Tony Hung,Matthew Troyer,Peter Chin,Jeff M. Harris,Anna Bakardjiev

Molecular Genetics and Metabolism(2023)

引用 1|浏览5
暂无评分
摘要
JC virus (JCV) is a member of the Polyomaviridae family and is associated to a severe disease known as progressive multifocal leukoencephalopathy, PML, which is gradually increasing in incidence as an opportunistic infection among AIDS patients. The present study aimed to investigate the occurrence of JCV among HIV-1 carriers including their types and molecular subtypes and the possible association with disease. Urine samples from 66 HIV-1 infected subjects were investigated for the presence of the virus by amplifying VP1 (215 bp) and IG (610 bp) regions using the polymerase chain reaction. JCV was detected in 32% of the samples. The results confirmed the occurrence of type B (subtype Af2); in addition, another polyomavirus, BKV, was also detected in 1.5% of samples of the HIV-1 infected subjects. Apparently, there was no significant difference between mono- (HIV-1 only) and co-infected (HIV-1/JCV) subjects regarding their TCD4+/TCD8+ lymphocyte counts or HIV-1 plasma viral load. Self admitted seizures, hearing and visual loses were not significantly different between the two groups.
更多
查看译文
关键词
lysosomal dysfunction,neuronopathic mps ii,biomarkers
AI 理解论文
溯源树
样例
生成溯源树,研究论文发展脉络
Chat Paper
正在生成论文摘要