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

The Wnt/β-catenin signaling pathway mediates renal fibrosis in glycogen storage disease type Ib nephropathy.

Wnt/β-catenin信号通路介导Ib型糖原贮积症肾病中的肾纤维化

Pratap Kunal, Lee Cheol, Zhang Lisa, Chen Hung Dar, Arnaoutova Irina, Mansfield Brian C, Chou Janice Y

Liver-Directed Gene Therapy Mitigates Early Nephropathy in Murine Glycogen Storage Disease Type Ia.

肝脏靶向基因疗法可减轻小鼠糖原贮积症 Ia 型早期肾病

Lee Cheol, Pratap Kunal, Zhang Lisa, Chen Hung Dar, Arnaoutova Irina, Starost Matthew F, Mansfield Brian C, Chou Janice Y

Novel murine model provides insights into early-onset of kidney disease in glycogen storage disease type Ib.

新型小鼠模型为糖原贮积症 Ib 型早期肾脏疾病的发生提供了见解。

Pratap Kunal, Lee Cheol, Zhang Lisa, Chen Hung Dar, Arnaoutova Irina, Mansfield Brian C, Chou Janice Y

CRISPR/Cas9-based double-strand oligonucleotide insertion strategy corrects metabolic abnormalities in murine glycogen storage disease type-Ia

基于 CRISPR/Cas9 的双链寡核苷酸插入策略可纠正小鼠 Ia 型糖原贮积症的代谢异常

Samanta, Ananya; George, Nelson; Arnaoutova, Irina; Chen, Hung-Dar; Mansfield, Brian C; Hart, Christopher; Carlo, Troy; Chou, Janice Y

Gene therapy and genome editing for type I glycogen storage diseases

基因疗法和基因组编辑治疗I型糖原贮积症

Chou, Janice Y; Mansfield, Brian C

Correction of metabolic abnormalities in a mouse model of glycogen storage disease type Ia by CRISPR/Cas9-based gene editing

利用基于 CRISPR/Cas9 的基因编辑技术纠正 Ia 型糖原贮积症小鼠模型中的代谢异常

Arnaoutova, Irina; Zhang, Lisa; Chen, Hung-Dar; Mansfield, Brian C; Chou, Janice Y

Inflammation in Viral Vector-Mediated Ocular Gene Therapy: A Review and Report From a Workshop Hosted by the Foundation Fighting Blindness, 9/2020

病毒载体介导的眼部基因治疗中的炎症:回顾与报告(来自对抗失明基金会主办的研讨会,2020年9月)

Chan, Ying Kai; Dick, Andrew D; Hall, Sara Mary; Langmann, Thomas; Scribner, Curtis L; Mansfield, Brian C

An evolutionary approach to optimizing glucose-6-phosphatase-α enzymatic activity for gene therapy of glycogen storage disease type Ia

利用进化方法优化葡萄糖-6-磷酸酶-α酶活性,用于糖原贮积症Ia型基因治疗

Zhang, Lisa; Cho, Jun-Ho; Arnaoutova, Irina; Mansfield, Brian C; Chou, Janice Y

Glycogen storage disease type Ia mice with less than 2% of normal hepatic glucose-6-phosphatase-α activity restored are at risk of developing hepatic tumors

肝糖原贮积症 Ia 型小鼠,若肝脏葡萄糖-6-磷酸酶-α 活性恢复不足正常值的 2%,则有发生肝肿瘤的风险。

Kim, Goo-Young; Lee, Young Mok; Kwon, Joon Hyun; Cho, Jun-Ho; Pan, Chi-Jiunn; Starost, Matthew F; Mansfield, Brian C; Chou, Janice Y

Liver-directed gene therapy for murine glycogen storage disease type Ib

针对小鼠Ib型糖原贮积症的肝脏靶向基因治疗

Kwon, Joon Hyun; Lee, Young Mok; Cho, Jun-Ho; Kim, Goo-Young; Anduaga, Javier; Starost, Matthew F; Mansfield, Brian C; Chou, Janice Y