Abstract
Animal models are extremely valuable to help us understand the pathogenesis of neurodegenerative disorders and to find treatments for them. Since large animals are more like humans than rodents, they make good models to identify the important pathological events that may be seen in humans but not in small animals; large animals are also very important for validating effective treatments or confirming therapeutic targets. Due to the lack of embryonic stem cell lines from large animals, it has been difficult to use traditional gene targeting technology to establish large animal models of neurodegenerative diseases. Recently, CRISPR/Cas9 was used successfully to genetically modify genomes in various species. Here we discuss the use of CRISPR/Cas9 technology to establish large animal models that can more faithfully mimic human neurodegenerative diseases.
Highlights
Animal models are extremely valuable to help us understand the pathogenesis of neurodegenerative disorders and to find treatments for them
We focus on the utilization of CRISPR/Cas9 in establishing large animal models of neurodegenerative diseases
CRISPR/Cas9 makes a powerful tool for generating non-human primate or large animal models of neurodegenerative diseases that are caused by the loss of function of specific genes
Summary
Animal models are extremely valuable to help us understand the pathogenesis of neurodegenerative disorders and to find treatments for them. Identification of the genetic mutations for different neurodegenerative diseases has enabled the generation of a variety of transgenic mouse models via expression of mutant proteins. We have various mouse models of neurodegenerative diseases from the expression of mutant genes under different promoters or from using other transgenic approaches.
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