Gene carrier for treating or preventing bietti's crystalline dystrophy and application of gene carrier

A retinal pigment and gene carrier technology, which is used in the introduction of foreign genetic material, gene therapy, genetic engineering and other directions using vectors to achieve the effect of delaying retinal degradation
CN109136266AActive Publication Date: 2019-01-04深圳泓熙生物科技发展有限公司
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Patent Information

Authority / Receiving Office
CN · China
Patent Type
Applications(China)
Current Assignee / Owner
深圳泓熙生物科技发展有限公司
Publication Date
2019-01-04

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Abstract

The invention relates to a gene carrier for treating or preventing bietti's crystalline dystrophy (BCD) and application of the gene carrier. The gene carrier for treating or preventing bietti's crystalline dystrophy comprises a packaging plasmid and a carrier plasmid. The packaging plasmid is an AAV2 packaging plasmid where a gene sequence of anti-CD59 short-chain-polypeptide C9 is inserted; the carrier plasmid is an AAV carrier plasmid where a gene sequence of human cytochrome P450 superfamily protein CYP4V2, a gene sequence of anti-VEGF short-chain-polypeptide HRH and a starting sub sequenceare inserted. The AAV carrier can conduct fusion expression on C9 anti-CD59 short-chain-polypeptide to virus coat protein; coexpression of CYP4V2 and HRH can be achieved, protein function loss causedby CYP4V2 genic mutation in a RPE cell is compensated for, the normal function of the RPE cell can be effectively repaired, specificity antagonism of a vascular endothelial growth factor (VEGF) can be achieved, and retinal atrophy caused by choroid hyperplasia is delayed. The invention further application of the gene carrier to preparation of medicine for treating or preventing bietti's crystalline dystrophy (BCD).
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Description

technical field

[0001] The invention belongs to the technical field of genetic engineering, and relates to a gene carrier, in particular to a gene carrier for treating or preventing crystalline retinitis pigmentosa (Bietti's crystalline dystrophy, BCD) and its use. Background technique

[0002] Crystalline retinitis pigmentosa (Bietti's crystalline dystrophy, BCD) is a hereditary retinal degenerative eye disease. Patients generally develop night blindness in adolescence; as the disease progresses, yellow-white crystalline lipid crystals appear in the cornea and fundus, accompanied by visual field loss; in the advanced stage of the disease, the patient's retina shrinks and eventually becomes blind1. The disease is a rare disease in the West, but in Asia, especially in China, it has a relatively high incidence (1 / 20000) (Hu, D.-N. (1983). Ophthalmic genetics in China. Ophthalmic Paediatrics and Genetics, 2 (1), 39-45.). Unfortunately, there is currently no effective treatmen...

Claims

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