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AAV (adeno-associated virus) virion with mutated capsid and application of AAV virion

A virosome and virus technology applied in the field of genetic engineering to achieve high transduction efficiency

Active Publication Date: 2019-06-18
苏州吉脉基因药物生物科技有限公司
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  • Description
  • Claims
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Problems solved by technology

[0005] However, there is no report on the efficient targeting of recombinant AAV virions to retinal Müller cells and its application in Müller cell gene therapy for retinopathy.

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  • AAV (adeno-associated virus) virion with mutated capsid and application of AAV virion
  • AAV (adeno-associated virus) virion with mutated capsid and application of AAV virion
  • AAV (adeno-associated virus) virion with mutated capsid and application of AAV virion

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Embodiment 1

[0077] This embodiment relates to an AAV variant that efficiently and specifically transduces Müller cells. The specific construction method includes the following steps:

[0078] (1) Preparation of recombinant plasmids (see Figure 1A-Figure 1D ):

[0079] A. Preparation of pFastbacdual-inCap6S663L

[0080] According to the literature (Chen H.Intron splicing-mediated expression of AAV Rep andCap genes and production of AAV vectors in insect cells.Mol Ther, 2008,16(5):924-930), the intron sequence was synthesized, and the intron sequence was synthesized by the fusion PCR method. It was inserted between bases 25 and 26 of the Cap6 sequence, and the PCR product was digested with BamHI and XbaI and ligated into the multiple cloning site (MCS) of the pFastbacdual plasmid to obtain pFastbacdual-inCap6. Further, using the pFastbacdual-inCap6 plasmid as a template, the gene sequence encoding the 663rd serine of Cap6 protein was subjected to site-directed mutation by fusion PCR metho...

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Abstract

The invention discloses an AAV (adeno-associated virus) virion with a mutated capsid and an application of the AAV virion. The AAV virion with the mutated capsid has mutated AAV6 capsid protein, and endows enhanced retina Muller (shown in the description) cells with infectivity; compared with corresponding parent AAV6 capsid protein, 663-poisiton serine in the amino acid sequence of the mutated AAV6 capsid protein is mutated into leucine; pharmaceutical composition contains the AAV virion and a pharmaceutically acceptable excipient. By means of site-directed mutation of amino acid of encoded AAV6 capsid, an AAV vector for specifically and efficiently transducing the Muller (shown in the description) cells is obtained, and is suitable for carrying exogenous therapeutic gene transduced Muller (shown in the description) cells to treat retinopathy.

Description

technical field [0001] The present invention relates to a recombinant AAV virion, in particular to a method for single-point amino acid mutations in selected adeno-associated virus (AAV) sequences to confer targeting of retinal Müller cells, and in the delivery of exogenous therapeutic gene transduction The application of Müller cells 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 (Adeno-assoc...

Claims

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Application Information

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IPC IPC(8): C12N7/00C12N15/35C12N15/63A61K48/00A61P9/10
Inventor 邵嘉红赵晓明谈鹏程吴相荀婷君陆阳
Owner 苏州吉脉基因药物生物科技有限公司
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