Local transgene expression and whole‐body transgenesis to model brain diseases in nonhuman primate

Abstract

Animal model is an essential tool in the life sciences research, notably in understanding the pathogenesis of the diseases and for further therapeutic intervention success. Rodents have been the most frequently used animals to model human disease since the establishment of gene manipulation technique. However, they remain inadequate to fully mimic the pathophysiology of human brain disease, partially due to huge differences between rodents and humans in terms of anatomy, brain function, and social behaviors. Nonhuman primates are more suitable in translational perspective. Thus, genetically modified animals have been generated to investigate neurologic and psychiatric disorders. The classical transgenesis technique is not efficient in that model; so, viral vector‐mediated transgene delivery and the new genome‐editing technologies have been promoted. In this review, we summarize some of the technical progress in the generation of an ad hoc animal model of brain diseases by gene delivery and real transgenic nonhuman primate.

Spatially resolved gene regulatory and disease-related vulnerability map of the adult Macaque cortex

“…4 College of Life Sciences, University of Chinese Academy of Sciences, Beijing 100049, China. 5 School of Biology and Biological Engineering, South China University of Technology, Guangzhou 510006, China. 6 Hematology and Cell Therapy Unit, IRCCS-Istituto Tumori "Giovanni Paolo II".…”

“…With a similar neocortex organization in monkey and human brain, the monkey offers a unique model for studying features of human neurodevelopment and neuropsychiatric diseases [1][2][3] . The cynomolgus monkey (Macaca fascicularis) is one of the most studied non-human primates (NHP) in neuroscience and medicine 4,5 . Recent advances in transgenesis and genome-editing technologies have led to successful development of new cynomolgus monkey genetic models to study human neurological disorders [6][7][8] , making this species an excellent experimental NHP model for studying higher order brain function.…”

Spatially resolved gene regulatory and disease vulnerability map of the adult Macaque cortex

“…The cynomolgus monkey (Macaca fascicularis) is one of the most studied non-human primates (NHP) in neuroscience and medicine (6,7). Recent advances in transgenesis and genome-editing technologies have led to successful development of 5 new cynomolgus monkey models to study genetic human neurological disorders (8)(9)(10), making this species an excellent experimental NHP model for brain researchers.…”

Hydrogels in Gene Delivery Techniques for Regenerative Medicine and Tissue Engineering

“…[ 90 ] Gene delivery can provide rather long‐ or short‐term expression of a transgene at a specific site, resulting in localized or systemic effects. [ 91 ] Hydrogels combined with gene carriers can act in vivo and in vitro experiments by different mechanisms ( Figure 3 ) and have numerous advantages when used as scaffolds for gene delivery systems. In this section, we will introduce six common gene delivery vectors ( Figure 4 ).…”