Construction method and application of retinitis pigmentosa animal model
A retinal pigment and model technology, applied in the field of biomedicine, can solve the problems of unclear pathogenic mechanism of mutant genes, lack of etiology and pathological mechanism, obstacles, etc.
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[0060] After extensive and in-depth research, the inventors discovered for the first time that there is a c.262dupC mutation in CTSD gene unexpectedly in RP patients. Even more unexpectedly, when the virus vector rAAV / CTSD-MUT was introduced into the retina, the mice developed retinitis pigmentosa-like pathological manifestations after 4-8 weeks, suggesting that the CTSD mutant protein may have retinal toxicity, so a non-retinopathy-like pathology of retinitis pigmentosa can be established. Preparation of human mammalian models. On this basis, the inventors have completed the present invention.
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