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Adeno-associated virus virions of the exophilic retina and their applications

A technology of the outer retina, virions, applied in the field of genetic engineering

Active Publication Date: 2021-08-06
CENT SOUTH UNIV
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

[0005] However, there have not been any reports on the efficient targeting of recombinant AAV virions to outer retinal cells to improve the transduction efficiency of retinal outer layer cells

Method used

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  • Adeno-associated virus virions of the exophilic retina and their applications
  • Adeno-associated virus virions of the exophilic retina and their applications
  • Adeno-associated virus virions of the exophilic retina and their applications

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Embodiment 1

[0072] The present invention relates to an AAV vector of outer-philic retina, and the specific construction method comprises the following steps:

[0073] (1) Preparation of recombinant plasmids:

[0074] A. Preparation of pFastbacdual-inCap9-tYF

[0075] Site-directed mutagenesis by fusion PCR requires two rounds of PCR reactions. In the first round of PCR, the Cap gene in the pFastbacdual-inCap9 plasmid is used as a template. The first round of PCR involves two reaction systems, in which the first sequence The 3' primer of the first sequence and the 5' primer of the second sequence have a complementary region; then all the products of the first round of PCR are added to a reaction system as a template, the 5' primer of the first sequence and the 3' primer of the last sequence The 'primer is used as a primer for the second amplification to form a fusion sequence. After the second round of PCR products were purified by gel cutting and DNA purification kit, they were digested...

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Abstract

The invention discloses an adeno-associated virus virion of outer-phile retina and application thereof. The retinal-exotropic adeno-associated virus virion has an amino acid mutation in the AAV9 capsid protein that confers enhanced infectivity of retinal-exotropic cells. Wherein, relative to the corresponding parental AAV9 capsid protein, tyrosines at positions 274, 446 and 731 in the amino acid sequence of the mutant AAV9 capsid protein are mutated to phenylalanine. The pharmaceutical composition comprises the adeno-associated virus virion of the exophilic retina and a pharmaceutically acceptable excipient. The invention obtains the adeno-associated virus mutant of the outer layer retina by performing amino acid site-directed mutation on the coding AAV9 virus capsid, and is suitable for exogenous therapeutic gene transduction outer layer retinal cells to treat retinopathy.

Description

technical field [0001] The present invention relates to a recombinant AAV virion, and in particular to a method of making amino acid mutations in selected adeno-associated virus (AAV9) sequences to confer its exotropic retinal affinity, and to transducing exogenous therapeutic genes with exogenous The application of the multilayer retinal cell in treating retinopathy belongs to the technical field of genetic engineering. Background technique [0002] Retinal diseases are one of the main causes of blindness. Common ones include macular degeneration, diabetic retinopathy, glaucoma, and hereditary retinopathy. Among them, most of the lesions are caused by gene mutations, protein failure or overexpression, which lead to the death of visual cells and eventually blindness. Through gene therapy, the correct gene can be mediated or the abnormal gene can be knocked out, and the normal expression can be restored, thereby restoring visual function. [0003] Adeno-associated virus (AA...

Claims

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Application Information

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Patent Type & Authority Patents(China)
IPC IPC(8): A61K48/00C12N7/00C07K14/015C12N15/35A61P27/02
CPCA61K48/0025A61P27/02C07K14/005C12N7/00C12N2750/14121
Inventor 李文生王雪
Owner CENT SOUTH UNIV