Abstract
Gene editing tools are essential for uncovering how genes mediate normal brain–behavior relationships and contribute to neurodegenerative and neuropsychiatric disorders. Recent progress in gene editing technology now allows neuroscientists unprecedented access to edit the genome efficiently. Although many important tools have been developed, here we focus on approaches that allow for rapid gene editing in the adult nervous system, particularly CRISPR/Cas9 and anti-sense nucleotide-based techniques. CRISPR/Cas9 is a flexible gene editing tool, allowing the genome to be manipulated in diverse ways. For instance, CRISPR/Cas9 has been successfully used to knockout genes, knock-in mutations, overexpress or inhibit gene activity, and provide scaffolding for recruiting specific epigenetic regulators to individual genes and gene regions. Moreover, the CRISPR/Cas9 system may be modified to target multiple genes at one time, affording simultaneous inhibition and overexpression of distinct genetic targets. Although many of the more advanced applications of CRISPR/Cas9 have not been applied to the nervous system, the toolbox is widely accessible, such that it is poised to help advance neuroscience. Anti-sense nucleotide-based technologies can be used to rapidly knockdown genes in the brain. The main advantage of anti-sense based tools is their simplicity, allowing for rapid gene delivery with minimal technical expertise. Here, we describe the main applications and functions of each of these systems with an emphasis on their many potential applications in neuroscience laboratories.
Highlights
Gene editing tools are essential for uncovering how genes mediate normal brain–behavior relationships and contribute to neurodegenerative and neuropsychiatric disorders
Many important tools have been developed, here we focus on approaches that allow for rapid gene editing in the adult nervous system, CRISPR/Cas9 and anti-sense nucleotide-based techniques
We focus on removing the need for viruses/mouse models by highlighting the advances made in anti-sense nucleotides, mainly anti-sense oligonucleotides (ASOs) and Morpholinos, which can be directly added to the brain without any delivery system, to effect gene regulation
Summary
Advanced In vivo Use of CRISPR/Cas and Anti-sense DNA Inhibition for Gene Manipulation in the. Many important tools have been developed, here we focus on approaches that allow for rapid gene editing in the adult nervous system, CRISPR/Cas and anti-sense nucleotide-based techniques. In addition to elucidating the roles genes play in normal regulation of cognitive and affective functions, this understanding is key for developing novel and effective therapeutic interventions for brain disorders (Mamdani et al, 2013; Bae et al, 2015; Szyf, 2015). Achieving this goal requires the ability to manipulate (both increase and decrease) gene expression. We will discuss the latest advances in anti-sense oligonucleotide approaches for gene knockdown because of their high level of accessibility and ease with which they can be applied across neuroscience laboratories with minimal requirements for in-house design
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