Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier

被引：10
作者：
Critchley, Bethan J. [1 ]
Gaspar, H. Bobby [1 ,2 ]
Benedetti, Sara [1 ,3 ]
机构：
[1] UCL Great Ormond St Inst Child Hlth, Zayed Ctr Res, Infect Immun & Inflammat Res & Teaching Dept, London WC1N 1DZ, England
[2] Orchard Therapeut Ltd, London EC4N 6EU, England
[3] NIHR Great Ormond St Hosp Biomed Res Ctr, London, England
来源：
MOLECULAR THERAPY | 2023年 / 31卷 / 03期
关键词：
ENZYME REPLACEMENT THERAPY; STEM-CELL TRANSPLANTATION; NEURONAL CEROID-LIPOFUSCINOSIS; MUCOPOLYSACCHARIDOSIS TYPE-II; INTRACEREBROVENTRICULAR GENE-THERAPY; TRANSFERRIN RECEPTOR ANTIBODY; DELAYS NEUROLOGICAL DISEASE; PRECLINICAL MOUSE MODEL; ALPHA-L-IDURONIDASE; CANINE MODEL;
D O I：
10.1016/j.ymthe.2022.11.015
中图分类号：
Q81 [生物工程学（生物技术）]; Q93 [微生物学];
学科分类号：
071005 ; 0836 ; 090102 ; 100705 ;
摘要：
Lysosomal storage diseases (LSDs) are multisystem inherited metabolic disorders caused by dysfunctional lysosomal activity, resulting in the accumulation of undegraded macromolecules in a variety of organs/tissues, including the central nervous sys-tem (CNS). Treatments include enzyme replacement therapy, stem/progenitor cell transplantation, and in vivo gene therapy. However, these treatments are not fully effective in treating the CNS as neither enzymes, stem cells, nor viral vectors efficiently cross the blood-brain barrier. Here, we review the latest ad-vancements in improving delivery of different therapeutic agents to the CNS and comment upon outstanding questions in the field of neurological LSDs.
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收藏
页码：657 / 675
页数：19
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