Method for preparing retinitis pigmentosa non-human mammal model

A non-human mammal and retinal pigment technology, applied in the fields of molecular biology and biomedicine, can solve the problems of complex pathological background, cognitive decline, and difficulty in screening and research of animals, and achieve the effect of simple pathological background
CN111979241BActive Publication Date: 2021-10-01SHANGHAI FIRST PEOPLES HOSPITAL
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Patent Information

Authority / Receiving Office
CN Β· China
Patent Type
Patents(China)
Current Assignee / Owner
SHANGHAI FIRST PEOPLES HOSPITAL
Publication Date
2021-10-01

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Abstract

The invention relates to a method for preparing a non-human mammalian model of retinitis pigmentosa. The method comprises replacing the endogenous ctsd gene on one of the homologous chromosomes of the animal with the h-CTSD* gene; the h-CTSD* gene is a human ctsd gene with a c.262dupC site mutation. This application first discovered that the h-CTSD* gene is related to RP, and established a mouse animal model in which the h-CTSD* gene was knocked in. The invention provides a convenient, reliable and economical means for studying the relationship between CTSD mutation and retinitis pigmentosa and its pathogenic mechanism, and provides a reliable theoretical basis for the research. More importantly, the animal does not show symptoms of neurological diseases such as spasms, neuronal ceroid lipofuscin deposition, ataxia, etc., except for RP, and the pathological background is simpler, which is very suitable for the construction of RP animal models .
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Description

technical field

[0001] The invention relates to the technical fields of molecular biology and biomedicine, in particular to a method for preparing a non-human mammalian model of retinitis pigmentosa. Background technique

[0002] CRISPR / Cas9 technology is a new gene editing technology developed in recent years. It is developed from the adaptive immune defense formed by archaea and bacteria under long-term selective pressure, which can effectively resist the invasion of foreign DNA. The immune system consists of three components: Cas9 protein, CRISPR RNAs (crRNAs) and trans-activating crRNAs (tracrRNA). In the process of immune defense, guide crRNA guides Cas9 protein to target foreign genes and cut them. Accordingly, people have modified crRNA-tracrRNA in vitro to obtain a sgRNA that can recognize the NGG PAM (protosp aceradjacentmotif) sequence and the target gene. At the same time, it can bind to the Cas9 protein and guide it to cut at 3 to 8 bp upstream of the PAM to form...

Claims

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