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首页> 外文期刊>Expert opinion on biological therapy >Gene therapy for lysosomal storage disorders.
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Gene therapy for lysosomal storage disorders.

机译：溶酶体贮积症的基因治疗。

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INTRODUCTION: Lysosomal storage disorders (LSDs) encompass more than 50 distinct diseases, caused by defects in various aspects of lysosomal function. Neurodegeneration and/or dysmyelination are the hallmark of roughly 70% of LSDs. Gene therapy represents a promising approach for the treatment of CNS manifestations in LSDs, as it has the potential to provide a permanent source of the deficient enzyme, either by direct injection of vectors or by transplantation of gene-corrected cells. In this latter approach, the biology of neural stem/progenitor cells and hematopoietic cells might be exploited. 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.
机译：简介：溶酶体贮积症（LSD）包含50多种由溶酶体功能各方面的缺陷引起的疾病。神经变性和/或髓鞘异常是约70％LSD的标志。基因疗法代表了一种治疗LSD中枢神经系统表现的有前途的方法，因为它有可能通过直接注射载体或通过基因校正的细胞移植来提供缺陷酶的永久来源。在后一种方法中，可以利用神经干/祖细胞和造血细胞的生物学功能。覆盖的领域：在直至2011年3月的大量文献基础上，作者回顾并讨论了以靶向中枢神经系统为目标的新型基因治疗策略的进展，关键方面和主要挑战，特别是对直接脑内基因的关注神经干/祖细胞的递送和移植。专家意见：实施具有特定嗜性，调节的转基因表达，低免疫原性和低遗传毒性风险的病毒载体递送系统，以及改善要移植的相关细胞类型的分离和操作，是该领域的基本挑战。同样，可能需要组合策略来在神经系统受累的LSD中实现完全纠正。

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