Novel siRNA delivery strategy: a new "strand" in CNS translational medicine?

被引：14

作者：

Gherardini, Lisa ^{[1
]}

Bardi, Giuseppe ^{[2
]}

Gennaro, Mariangela ^{[3
]}

Pizzorusso, Tommaso ^{[3
,4
]}

机构：

[1] CNR, Inst Clin Physiol, Siena, Italy

[2] Ist Italiano Tecnol, Ctr MicroBioRobot SSSA, I-56025 Pontedera, Italy

[3] CNR, Inst Neurosci, I-56124 Pisa, Italy

[4] Univ Florence, Dept Neurosci Psychol Drug Res & Child Hlth NEURO, Florence, Italy

来源：

CELLULAR AND MOLECULAR LIFE SCIENCES | 2014年 / 71卷 / 01期

关键词：

Neurodegeneration; Gene therapy; RNA interference; Nanoparticles; SMALL-INTERFERING RNA; CENTRAL-NERVOUS-SYSTEM; NEURONAL CEROID-LIPOFUSCINOSIS; ALZHEIMERS-DISEASE THERAPY; HUMAN GLIOMA-CELLS; GENE-THERAPY; IN-VIVO; PARKINSONS-DISEASE; MUTANT SOD1; HUNTINGTONS-DISEASE;

D O I：

10.1007/s00018-013-1310-8

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

070307 [化学生物学]; 071010 [生物化学与分子生物学];

摘要：

RNA interference has been envisaged as a powerful tool for molecular and clinical investigation with a great potential for clinical applications. In recent years, increased understanding of cancer biology and stem cell biology has dramatically accelerated the development of technology for cell and gene therapy in these areas. This paper is a review of the most recent report of innovative use of siRNA to benefit several central nervous system diseases. Furthermore, a description is made of innovative strategies of delivery into the brain by means of viral and non-viral vectors with high potential for translation into clinical use. Problems are also highlighted that might hamper the transition from bench to bed, analyzing the lack of reliable preclinical models with predictive validity and the lack of effective delivery systems, which are able to overcome biological barriers and specifically reach the brain site of action.

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页码：1 / 20

页数：20

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