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Gene therapy carrier for treating retinitis pigmentosa diseases

A technology of viral vectors and vectors, applied in the field of genetic engineering, can solve problems such as complex pathogenic mechanisms

Active Publication Date: 2020-05-08
SHANGHAI LANGSHENG BIOTECHNOLOGY CO LTD
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

Due to its significant genetic heterogeneity and complex pathogenic mechanism, there is no clear treatment for this disease

Method used

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  • Gene therapy carrier for treating retinitis pigmentosa diseases
  • Gene therapy carrier for treating retinitis pigmentosa diseases
  • Gene therapy carrier for treating retinitis pigmentosa diseases

Examples

Experimental program
Comparison scheme
Effect test

Embodiment 1

[0176] Example 1 Sequence Optimization

[0177] In this example, based on the amino acid sequence of PROM1 protein (SEQ ID No.: 3) and the natural coding sequence (SEQ ID No.: 2), the inventors optimized the coding sequence. Specifically, the inventors optimized sequence fragments that affect gene expression, including but not limited to: codon usage bias, elimination of secondary structures that are unfavorable to expression (such as hairpin structures), changes in GC content, CpG II Nucleotide content, secondary structure of mRNA, cryptic splice sites, early polyadenylation sites, internal ribosome entry and binding sites, negative CpG islands, regions of RNA instability, repetitive sequences (direct repeats , inverted repeats, etc.) and restriction sites that may affect cloning. Through analysis and test screening, a specially optimized DNA coding sequence as shown in SEQ ID NO.:1 is finally obtained.

[0178] Such as figure 1 As shown, the recombinant adeno-associated v...

Embodiment 2

[0181] 1. Experimental grouping:

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PUM

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Abstract

The invention relates to a gene therapy carrier for treating retinitis pigmentosa diseases. Specifically, a targeted special optimal design is performed on a PROM1 gene coding sequence, so that a nucleotide sequence which is especially suitable for efficiently expressing PROM1 protein in cells (particularly photosensory cells) in lactation animals (such as people) is obtained, a recombinant AAV virus expressing normal human derived PROM1 protein is constructed, and the validity of the recombinant AAV virus in a PROM1 knockout mouse model is proved. The experiment result indicates that comparedwith the expression quantity of a coding sequence which is not optimized, the expression quantity of a PROM1 coding sequence (SEQ ID NO.:1) after special optimization is notably increased by 3 timesor above, so that the gene therapy carrier is extremely suitable for expression in the cells of the lactation animals (especially people), and can effectively treat eye diseases of the retinitis pigmentosa diseases and the like.

Description

technical field [0001] The invention relates to the technical field of genetic engineering, in particular to a gene therapy carrier for treating retinitis pigmentosa disease. Background technique [0002] Retinitis pigmentosa (RP) is a group of diseases characterized by hereditary, progressive photoreceptor cell loss and eventually retinal degeneration and atrophy, accompanied by dystrophic degeneration of retinal pigment epithelium (RPE) cells, reported in the literature The prevalence of RP is about 1 / 4000 worldwide, and more than 1.5 million patients suffer from progressive visual impairment. Lesions usually begin at the equator of the retina and progress toward the macula and fovea. Its main manifestations are night blindness, progressive peripheral vision loss, etc., and the loss of vision due to the death of photoreceptor cells. In the fundus, the retinal blood vessels were thinned and narrowed like a white line, and the atrophy-like changes in the macular area were ...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): C12N15/12C12N15/864C12N5/10A61K48/00A61K38/17A61P27/02
CPCC07K14/47C12N15/86A61K48/005A61K38/1709A61P27/02C12N2750/14143C07K14/70596C12N2800/22A01K2217/075A01K2227/105A01K2267/0306A61K38/00
Inventor 罗学廷孙晓东
Owner SHANGHAI LANGSHENG BIOTECHNOLOGY CO LTD
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