南方科技大学知识苑(SUSTech KC): A review on Gaucher disease: therapeutic potential of Î²-glucocerebrosidase-targeted mRNA/saRNA approach

题名	A review on Gaucher disease: therapeutic potential of Î²-glucocerebrosidase-targeted mRNA/saRNA approach
作者	Feng, Shunping1 ; Rcheulishvili, Nino1,2 ; Jiang, Xiaoming 3; Zhu, Pan 3; Pan, Xuehua 4; Wei, Meilan1 ; Wang, Peng George1 ; Ji, Yang1 ; Papukashvili, Dimitri1,2
通讯作者	Wang, Peng George; Ji, Yang; Papukashvili, Dimitri
发表日期	2024
DOI	10.7150/ijbs.87741
发表期刊	INTERNATIONAL JOURNAL OF BIOLOGICAL SCIENCES 影响因子和分区
ISSN	1449-2288
卷号	20 期号:6
摘要	Gaucher disease (GD), a rare hereditary lysosomal storage disorder, occurs due to a deficiency in the enzyme beta-glucocerebrosidase (GCase). This deficiency leads to the buildup of substrate glucosylceramide (GlcCer) in macrophages, eventually resulting in various complications. Among its three types, GD2 is particularly severe with neurological involvements. Current treatments, such as enzyme replacement therapy (ERT), are not effective for GD2 and GD3 due to their inability to cross the blood-brain barrier (BBB). Other treatment approaches, such as gene or chaperone therapies are still in experimental stages. Additionally, GD treatments are costly and can have certain side effects. The successful use of messenger RNA (mRNA)-based vaccines for COVID-19 in 2020 has sparked interest in nucleic acid-based therapies. Remarkably, mRNA technology also offers a novel approach for protein replacement more protein at lower doses. This review aims to explore the potential of a cost-effective mRNA/saRNA-based approach for GD therapy. The use of GCase-mRNA/saRNA as a protein replacement therapy could offer a new and promising direction for improving the quality of life and extending the lifespan of individuals with GD.
关键词	Gaucher disease mRNA saRNA GlcCer GCase lysosomal storage disease ERT SRT protein replacement therapy
相关链接	[来源记录]
收录类别	SCI
语种	英语
学校署名	第一 ; 通讯
资助项目	National Natural Science Foundation of China[82273841]
WOS研究方向	Biochemistry & Molecular Biology ; Life Sciences & Biomedicine - Other Topics
WOS类目	Biochemistry & Molecular Biology ; Biology
WOS记录号	WOS:001201419700003
出版者	IVYSPRING INT PUBL
来源库	Web of Science
引用统计	被引频次[WOS]：3
成果类型	期刊论文
条目标识符	http://sustech.caswiz.com/handle/2SGJ60CL/788541
专题	南方科技大学医学院_药理学系南方科技大学医学院
作者单位	1.Southern Univ Sci & Technol, Sch Med, Dept Pharmacol, Shenzhen 518000, Peoples R China 2.Chinese Acad Sci, Inst Microbiol, Beijing 100101, Peoples R China 3.Cheerland Biomed, Shenzhen, Peoples R China 4.Shenzhen Pengbo Biotech Co Ltd, Shenzhen, Peoples R China
第一作者单位	药理学系; 南方科技大学医学院
通讯作者单位	药理学系; 南方科技大学医学院
第一作者的第一单位	药理学系; 南方科技大学医学院
推荐引用方式 GB/T 7714	Feng, Shunping,Rcheulishvili, Nino,Jiang, Xiaoming,et al. A review on Gaucher disease: therapeutic potential of Î²-glucocerebrosidase-targeted mRNA/saRNA approach[J]. INTERNATIONAL JOURNAL OF BIOLOGICAL SCIENCES,2024,20(6).
APA	Feng, Shunping.,Rcheulishvili, Nino.,Jiang, Xiaoming.,Zhu, Pan.,Pan, Xuehua.,...&Papukashvili, Dimitri.(2024).A review on Gaucher disease: therapeutic potential of Î²-glucocerebrosidase-targeted mRNA/saRNA approach.INTERNATIONAL JOURNAL OF BIOLOGICAL SCIENCES,20(6).
MLA	Feng, Shunping,et al."A review on Gaucher disease: therapeutic potential of Î²-glucocerebrosidase-targeted mRNA/saRNA approach".INTERNATIONAL JOURNAL OF BIOLOGICAL SCIENCES 20.6(2024).