Method for preparing Bietti Crystalline Dystrophy (BCD) non-human mammal model and application thereof

Abstract

The invention relates to the technical field of molecular biology and biomedicine, and particularly, relates to a method for preparing a Bietti Crystalline Dystrophy (BCD) non-human mammal model and application thereof. The method comprises: knocking out a first exon in an animal CYP4V3 gene knockout transcript by using a Crispr-Cas system. The animal model prepared by the method generates typicalyellow-white crystal depositions at fundi at the age of 7 months, while an existing model can generate typical crystal depositions at least at the age of 12 months; the model is beneficial for developing experiment research as soon as possible in the time dimension; meanwhile, the animal model generates the outer nuclera layer atrophy corresponding to extinguishment of a and b waves in electroretinogram (ERG) at the age of 12 months in the natural course, while the existing model does not generate the outer nuclera layer atrophy in the followed course; and the model simulates decline of a visual function of a BCD patient better, and is more beneficial for exploring the pathological mechanism of BCD.

Description

technical field [0001] The invention relates to the technical fields of molecular biology and biomedicine, in particular to a method for preparing a Bietti crystal-like atrophy non-human mammalian model and its application. Background technique [0002] Bietti crystalline dystrophy (BCD) is a hereditary retinopathy with autosomal recessive inheritance. It is more common in Asian populations, especially in China and Japan. The disease has characteristic fundus manifestations: yellow-white crystal deposits at the posterior pole of the retina accompanied by retinal pigment epithelium (RPE) cell atrophy, pigment changes, and choroidal atrophy. The onset of the disease usually occurs between the ages of 20 and 40. With the progressive changes of night blindness, vision loss and visual field defects, their lives will be gradually affected. Existing BCD model animals have all introduced exogenous genes into the model cell genome, and the impact of exogenous genes on the animal ge...

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Problems solved by technology

Existing BCD model animals have introduced exogenous genes into the model cell genome, and the impact of exogenous genes on the animal genome is immeasurable

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