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Producer T cells: Using genetically engineered T cells as vehicles to generate and deliver therapeutics to tumors.产生性T细胞：利用基因工程改造的T细胞作为载体，生成治疗药物并将其递送至肿瘤部位。
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Nanomedicine in the diagnosis and therapy of neurodegenerative disorders.纳米医学在神经退行性疾病诊断与治疗中的应用
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本文引用的文献

1
Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice.异染性脑白质营养不良的基因治疗可逆转小鼠的神经损伤和缺陷。
J Clin Invest. 2006 Nov;116(11):3070-82. doi: 10.1172/JCI28873.
2
See no evil, hear no evil, do no evil: the lessons of immune privilege.非礼勿视，非礼勿听，非礼勿言：免疫豁免的启示。
Nat Immunol. 2006 Apr;7(4):354-9. doi: 10.1038/ni1328.
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Resting microglial cells are highly dynamic surveillants of brain parenchyma in vivo.静息态小胶质细胞是体内脑实质的高度动态监测者。
Science. 2005 May 27;308(5726):1314-8. doi: 10.1126/science.1110647. Epub 2005 Apr 14.
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The blood-brain barrier: bottleneck in brain drug development.血脑屏障：脑药物研发的瓶颈
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Gene therapy of metachromatic leukodystrophy.异染性脑白质营养不良的基因治疗。
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6
Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells.通过移植基因改造的造血干细胞纠正小鼠模型中的异染性脑白质营养不良。
J Clin Invest. 2004 Apr;113(8):1118-29. doi: 10.1172/JCI19205.
7
Bone marrow stem cells have the ability to populate the entire central nervous system into fully differentiated parenchymal microglia.骨髓干细胞有能力在整个中枢神经系统中增殖并分化为成熟的实质小胶质细胞。
FASEB J. 2004 Jun;18(9):998-1000. doi: 10.1096/fj.04-1517fje. Epub 2004 Apr 14.
8
Human CD34+ cells differentiate into microglia and express recombinant therapeutic protein.人类CD34+细胞分化为小胶质细胞并表达重组治疗性蛋白。
Proc Natl Acad Sci U S A. 2004 Mar 9;101(10):3557-62. doi: 10.1073/pnas.0306431101. Epub 2004 Feb 27.
9
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1.两名接受X连锁重症联合免疫缺陷（SCID-X1）基因治疗的患者中与LMO2相关的克隆性T细胞增殖。
Science. 2003 Oct 17;302(5644):415-9. doi: 10.1126/science.1088547.
10
Drug and gene delivery to the brain: the vascular route.药物和基因向大脑的递送：血管途径。
Neuron. 2002 Nov 14;36(4):555-8. doi: 10.1016/s0896-6273(02)01054-1.

小胶质细胞：中枢神经系统基因治疗的细胞载体。

Microglia: a cellular vehicle for CNS gene therapy.

作者信息

Neumann Harald

机构信息

Neural Regeneration Unit, Institute of Reconstructive Neurobiology, University of Bonn, and LIFE & BRAIN Center and Hertie Foundation, Bonn, Germany.

出版信息

J Clin Invest. 2006 Nov;116(11):2857-60. doi: 10.1172/JCI30230.

DOI:10.1172/JCI30230
PMID:17080190
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC1626126/
Abstract

Metachromatic leukodystrophy (MLD) is a lysosomal storage disease caused by deficiency of the enzyme arylsulfatase A (ARSA). MLD is characterized by progressive demyelination and neurological deficits. Treatment of MLD is still a challenge due to the fact that the blood-brain barrier is a major obstacle for most therapeutic substances. In this issue of the JCI, Biffi et al. report that genetically modified hematopoietic precursor cells transduced to overexpress ARSA and transplanted into mice with a targeted disruption of the murine Arsa gene (Arsa(-/-) mice) migrated into the CNS and cross-corrected brain ARSA deficiency (see the related article beginning on page 3070). Microglia served as a cellular vehicle to effectively deliver the enzyme to other brain cells while hepatocytes overexpressing ARSA increased plasma ARSA levels but failed to deliver ARSA into the CNS.

摘要

异染性脑白质营养不良（MLD）是一种由芳基硫酸酯酶A（ARSA）缺乏引起的溶酶体贮积病。MLD的特征是进行性脱髓鞘和神经功能缺损。由于血脑屏障是大多数治疗物质的主要障碍，MLD的治疗仍然是一个挑战。在本期《临床研究杂志》中，比菲等人报告称，经基因改造的造血前体细胞被转导以过表达ARSA，并移植到具有鼠源Arsa基因靶向破坏的小鼠（Arsa(-/-)小鼠）体内，这些细胞迁移到中枢神经系统并交叉纠正了脑内ARSA缺乏（见第3070页开始的相关文章）。小胶质细胞作为一种细胞载体，有效地将该酶传递给其他脑细胞，而过表达ARSA的肝细胞增加了血浆ARSA水平，但未能将ARSA传递到中枢神经系统。