Methyl CpG binding protein 2-based (MECP2-based) method for injecting virus into specific brain area of animal without therapeutic aim to perform gene editing

Abstract

The invention discloses a methyl CpG binding protein 2-based (MECP2-based) method for injecting a virus into a specific brain area of an animal without a therapeutic aim to perform gene editing. A cerebral stereotaxis injection pump is utilized to directly inject the virus into the specific brain area of a non-human primate; the injection volume is that 1 [mu]L of virus is injected per 1m<3>; andthe titer of the injected virus is 10*10<13> vg / mL. According to the method, the virus is directly injected into the specific brain area of the non-human primate to perform gene editing, and thus, a transgenic monkey model with the disease phenotype can be obtained in a short time, and the urgent research demand of an autism spectrum disorder (ASD) disease at the present stage is met.

Description

technical field [0001] The invention belongs to the field of transgenic technology and modeling of non-human primates, and specifically relates to a non-therapeutic method based on the MECP2 gene that injects viruses into specific brain regions of non-human primates for gene editing to construct non-human primates. Methods for animal-like ASD disease models. Background technique [0002] Autism Spectrum Disorder (ASD) is characterized by autism-like behavior, loss of motor control, irregular breathing, and skeletal problems. Adolescents with ASD cannot participate in the normal education process, which has a huge impact on their normal integration into society and their social function as adults. At present, there is no effective treatment for ASD diseases, so ASD has a serious negative impact on family happiness and harmonious development of society, and has been widely valued by all walks of life. [0003] In recent years, the prevalence of ADS has been rising and has be...

AI Technical Summary

Problems solved by technology

[0004] At present, there are only a few embryonic ASD macaque models for macaques, but (1) the genetic background of macaques varies greatly among individuals, and the problem of mosaicism in embryo transgenic technology cannot be solved, so it is still difficult for these macaques based on embryo transgenes to help us To observe and study the etiology and pathogenesis of ASD

(2) The natural sexual maturity time of macaques is 5-6 years, and the gestation period takes 6 months. They are singleton animals. Therefore, it is difficult to achieve genetically manipulated monkeys that are homozygous by mating transgenic macaques at this stage.

(3) Transgenic monkeys based on embryo transgenic technology have high mortality rate, low success rate, poor stability of transgenic gene retention, and extremely long onset time. At present, it is difficult to establish a stable transgenic monkey model

Even if it is successfully established, it is difficult for this monkey model to reproduce in large numbers and the maintenance cost is extremely high, so it is difficult to meet the urgent research needs at this stage

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