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Gene therapy of Leber genetics optic neuropathy

An optic nerve, treatment method technology, applied in the field of gene therapy, can solve problems such as poor curative effect, blindness of patients, and no effective treatment methods

Pending Publication Date: 2020-04-28
WUHAN NEUROPHTH BIOTECHNOLOGY LTD CO
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

At present, neurotrophic drugs or traditional Chinese medicine are often used for treatment, but the curative effect is not good.
Because there is no effective treatment, once the disease occurs, only a few can get better, and most patients will eventually go blind.

Method used

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  • Gene therapy of Leber genetics optic neuropathy
  • Gene therapy of Leber genetics optic neuropathy
  • Gene therapy of Leber genetics optic neuropathy

Examples

Experimental program
Comparison scheme
Effect test

Embodiment 1

[0187] Example 1 Preparation of recombinant adeno-associated virus

[0188] The present invention only takes the recombinant adeno-associated virus carrying the ND4 coding sequence as an example, but is not limited thereto.

[0189] Refer to the method in Example 1 of CN 102634527B to prepare recombinant adeno-associated virus rAAV2 / 2-ND4.

[0190] 1.1 Plasmid preparation: The fusion nucleic acid shown in SEQ ID NO.:6 was synthesized by Chengdu Qingke Zixi Biotechnology Co., Ltd. Amplify the full-length gene by PCR, form sticky ends on the fusion gene by EcoRI / SalI digestion, and embed the fusion gene into the adeno-associated virus vector pSNaV with EcoRI / SalI restriction site, namely pSNaV / rAAV2 / 2-ND4 (hereinafter abbreviated as pAAV2-ND4). The screening and identification steps of the recombinants are the same as those of CN102634527B, and the brief description is as follows: Take the LB plate cultured at 37°C, blue spots and white spots appear, and the white ones are rec...

Embodiment 2

[0197] Example 2 Gene therapy LHON pre-treatment, treatment and post-treatment treatment, and curative effect

[0198] 1. Specifications before treatment:

[0199] 1. Research site

[0200] This study is a clinical trial, and the genetic diagnosis was completed in the genetic diagnosis center of the hospital; the ophthalmic examination was completed with the same type of equipment in the eye examination room of Tongji Hospital; the whole body examination (part) was completed by designated personnel and designated equipment in the laboratory department; all operations were performed The operation is performed in the special laminar flow operation room of the ophthalmology department; the postoperative review and follow-up are completed by the specially designated personnel and equipment of the team.

[0201] 2. Clinical trial design

[0202] This study was a multicenter, prospective clinical trial. The subjects meet the diagnostic criteria of Leber hereditary optic neuropath...

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Abstract

The invention relates to gene therapy of Leber genetics optic neuropathy. It is accidentally found in the invention that administration of Medrol before and after gene therapy of ocular diseases (i.e., administration of recombinant adeno-associated virus) not only can remarkably improve the treatment effect, but also can effectively prevent and / or treat complications of gene therapy, such as selective cataract removal, uveitis, anterior chamber inflammation and vitreitis, and causes small side effects.

Description

technical field [0001] The invention relates to the field of gene therapy, in particular to gene therapy for Leber hereditary optic neuropathy. Background technique [0002] Leber's hereditary optic neuropathy (LHON) is a hereditary disease that mainly involves the fibers of the papillary macula, leading to degeneration of the optic nerve. It is a maternally inherited disease whose etiology and mechanism have not yet been elucidated. The year was named after the first German scholar Leber to describe its clinical features. LHON is relatively rare, clinically manifested as acute or subacute painless vision loss in both eyes at the same time or successively, accompanied by loss of central visual field and color vision disturbance. At present, neurotrophic drugs or traditional Chinese medicine are often used for treatment, but the curative effect is not good. Because there is no effective treatment, once the disease occurs, only a few can get better, and most patients will ev...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K48/00A61K38/16A61P27/02A61P27/06A61P9/10
CPCA61K38/16A61K48/0033A61K48/005A61P9/10A61P27/02A61P27/06A61K48/00C12N15/09
Inventor 李斌
Owner WUHAN NEUROPHTH BIOTECHNOLOGY LTD CO
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