Gene therapy for lysosomal storage disorders

被引：25
作者：
Gritti, Angela [1 ]
机构：
[1] Ist Sci San Raffaele, San Raffaele Telethon Inst Gene Therapy HSR TIGET, I-20132 Milan, Italy
来源：
EXPERT OPINION ON BIOLOGICAL THERAPY | 2011年 / 11卷 / 09期
关键词：
CNS; gene delivery; gene therapy; lysosomal storage disorders; viral vectors; NEURAL STEM-CELLS; CENTRAL-NERVOUS-SYSTEM; ADENOASSOCIATED VIRUS VECTOR; ENZYME REPLACEMENT THERAPY; BONE-MARROW-TRANSPLANTATION; NONHUMAN PRIMATE BRAIN; MOUSE MODEL; LONG-TERM; LENTIVIRAL VECTORS; IMMUNE-RESPONSES;
D O I：
10.1517/14712598.2011.582036
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：
Areas covered: Based on an extensive literature search up until March 2011, the author reviews and discusses the progress, the crucial aspects and the major challenges towards the development of novel gene therapy strategies aimed to target the CNS, with particular attention to direct intracerebral gene delivery and transplantation of neural stem/progenitor cells. Expert opinion: The implementation of viral vector delivery systems with specific tropism, regulated transgene expression, low immunogenicity and low genotoxic risk and the improvement in isolation and manipulation of relevant cell types to be transplanted, are fundamental challenges to the field. Also, combinatorial strategies might be required to achieve full correction in LSDs with neurological involvement.
引用
收藏
页码：1153 / 1167
页数：15
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