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Prog Neurobiol. 2018 Sep;168:69-85. doi: 10.1016/j.pneurobio.2018.04.008. Epub 2018 Apr 22.

In vivo methods for acute modulation of gene expression in the central nervous system.

Author information

1
Local Micro-environment and Brain Development Laboratory, Istituto Italiano di Tecnologia, via Morego, 30, 16163 Genova, Italy; Università degli Studi di Genova, Via Balbi, 5, 16126 Genova, Italy.
2
Local Micro-environment and Brain Development Laboratory, Istituto Italiano di Tecnologia, via Morego, 30, 16163 Genova, Italy; Bio@SNS, Scuola Normale Superiore, Piazza dei Cavalieri 7, 56126, Pisa, Italy.
3
Local Micro-environment and Brain Development Laboratory, Istituto Italiano di Tecnologia, via Morego, 30, 16163 Genova, Italy; DulbeccoTelethon Institute, Italy. Electronic address: laura.cancedda@iit.it.

Abstract

Accurate and timely expression of specific genes guarantees the healthy development and function of the brain. Indeed, variations in the correct amount or timing of gene expression lead to improper development and/or pathological conditions. Almost forty years after the first successful gene transfection in in vitro cell cultures, it is currently possible to regulate gene expression in an area-specific manner at any step of central nervous system development and in adulthood in experimental animals in vivo, even overcoming the very poor accessibility of the brain. Here, we will review the diverse approaches for acute gene transfer in vivo, highlighting their advantages and disadvantages with respect to the efficiency and specificity of transfection as well as to brain accessibility. In particular, we will present well-established chemical, physical and virus-based approaches suitable for different animal models, pointing out their current and future possible applications in basic and translational research as well as in gene therapy.

KEYWORDS:

Electroporation; In vivo genetic manipulations; Nanoparticles; Polymers; Sonoporation; Viruses

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