日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

AAV Gene Therapy for MPS IVA with Induction of Immune Tolerance via Oral Administration of Epitope Peptides of N-Acetylgalactosamine-6-sulfate Sulfatase

利用口服N-乙酰半乳糖胺-6-硫酸酯酶表位肽诱导免疫耐受,采用腺相关病毒(AAV)基因疗法治疗MPS IVA

Saikia, Sampurna; Ago, Yasuhiko; Nidhi, Fnu; Khan, Shaukat; Ma, Zhengyu; Tomatsu, Shunji

Neuraminidase 1 secondary deficiency contributes to CNS pathology in neurological mucopolysaccharidoses via brain protein hypersialylation

神经氨酸酶 1 继发性缺乏症通过脑蛋白过度唾液酸化作用导致神经系统粘多糖贮积症的中枢神经系统病变。

Xu, TianMeng; Heon-Roberts, Rachel; Moore, Travis; Dubot, Patricia; Pan, Xuefang; Guo, Tianlin; Cairo, Christopher W; Holley, Rebecca J; Bigger, Brian; Durcan, Thomas M; Levade, Thierry; Ausseil, Jerôme; Amilhon, Bénédicte; Gorelik, Alexei; Nagar, Bhushan; Khan, Shaukat; Tomatsu, Shunji; Sturiale, Luisa; Palmigiano, Angelo; Röckle, Iris; Thiesler, Hauke; Hildebrandt, Herbert; Garozzo, Domenico; Pshezhetsky, Alexey V

Identification of Surrogate Biomarkers for Mucopolysaccharidosis Type IVA

粘多糖贮积症IVA型替代生物标志物的鉴定

Ago, Yasuhiko; Khan, Shaukat; Klipner, Kimberly; Bradford, Allison; Tomatsu, Shunji

CRISPR/nCas9-Edited CD34+ Cells Rescue Mucopolysaccharidosis IVA Fibroblasts Phenotype.

CRISPR/nCas9 编辑的 CD34+ 细胞挽救粘多糖贮积症 IVA 成纤维细胞表型

Herreno-Pachón Angélica María, Leal Andrés Felipe, Khan Shaukat, Alméciga-Díaz Carlos Javier, Tomatsu Shunji

Evaluation of AAV vectors with tissue-specific or ubiquitous promoters in a mouse model of mucopolysaccharidosis type IVA

在IVA型粘多糖贮积症小鼠模型中评估具有组织特异性或普遍表达启动子的AAV载体

Khan, Shaukat A; Álvarez, Jose Victor; Nidhi, F N U; Benincore-Florez, Eliana; Tomatsu, Shunji

Neonatal gene therapy effectively prevents disease manifestations in a murine model of Mucopolysaccharidosis type I

新生儿基因疗法可有效预防小鼠I型粘多糖贮积症模型中的疾病表现。

De Ponti, Giada; Santi, Ludovica; Dina, Giorgia; Pievani, Alice; Donsante, Samantha; Riminucci, Mara; Corsi, Alessandro; Khan, Shaukat; Passerini, Laura; Annoni, Andrea; Gregori, Silvia; Crippa, Stefania; Biondi, Andrea; Quattrini, Angelo; Tomatsu, Shunji; Aiuti, Alessandro; Bernardo, Maria Ester; Serafini, Marta

Three-dimensional human mucopolysaccharidosis IVA chondrocyte culture reveals significant impairments in the lysosomal-mitochondrial crosstalk

三维人粘多糖贮积症IVA型软骨细胞培养揭示溶酶体-线粒体相互作用的显著缺陷

Leal, Andrés Felipe; Saikia, Sampurna; Khan, Shaukat A; Tomatsu, Shunji

Potential Health and Cost Impacts of a Point-of-Care Test for Neonatal Sepsis and Possible Serious Bacterial Infections in Infants: A Modeling Analysis in Two Settings

即时检测新生儿败血症和婴儿可能出现的严重细菌感染的潜在健康和成本影响:两种环境下的建模分析

Chevalier, Joshua M; Hansen, Megan A; Grantz, Kyra H; Gleeson, Birgitta; Blumel, Benjamin; Chuchu, Veronicah; Khan, Shaukat; Sigwebela, Ntombi; Chimhini, Gwendoline; Fitzgerald, Felicity; Ferreyra, Cecilia; Nichols, Brooke E

The impact and cost of a new rapid diagnostic test for school-based prevalence mapping and monitoring and evaluation surveys of schistosomiasis: A modelling study

一项关于新型快速诊断测试在学校血吸虫病流行率调查、监测和评估中的影响和成本的建模研究

Chevalier, Joshua M; Grantz, Kyra H; Girdwood, Sarah; Kepha, Stella; Ramos, Thierry; Nichols, Brooke E; Khan, Shaukat; Hingel, Sarah

Collagen Type II-Targeting Lentiviral Gene Therapy for Mucopolysaccharidosis IVA

针对II型胶原蛋白的慢病毒基因疗法治疗IVA型粘多糖贮积症

Celik, Betul; Saikia, Sampurna; Khan, Shaukat; Musini, Krishna Sai; Tomatsu, Shunji