Application of arglabin in preparation of drugs for Stargardt disease

A macular degeneration and application technology, applied in sensory diseases, drug combinations, pharmaceutical formulations, etc., to achieve the effects of high safety factor, high safety, and convenient administration methods

Inactive Publication Date: 2018-12-21
XIAMEN UNIV
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  • Abstract
  • Description
  • Claims
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  • Application of arglabin in preparation of drugs for Stargardt disease
  • Application of arglabin in preparation of drugs for Stargardt disease
  • Application of arglabin in preparation of drugs for Stargardt disease

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Embodiment

[0029] Abca4 gene knockout mice are mostly used in the development of therapeutic drugs for STGD1. However, unlike STGD1 patients, in single-gene knockout mice, the degeneration of the retina is slow, and the more obvious pathological phenotypes generally appear after 8 months of age, which is not consistent with the phenotype of STGD1 juvenile onset. Therefore, in 2008 the laboratory of Krzysztof Palczewski reported the production of Abca4- / - R8 - / - Double-knockout mice have obvious STGD1 pathological changes such as shortening of the outer segment of photoreceptor cells, thinning of the outer nuclear layer, and accumulation of A2E at the age of 4 months, which can be used as an animal model of STGD1 disease. This embodiment adopts Abca4 - / - R8 - / - Double knockout mice were performed.

[0030] Agalabi was dissolved in DMSO to make 25ng / μL stock solution. Choose 2 months old Abca4 - / - R8 - / - Double-knockout mice, 6 mice were used as the agaleb treatment group, the mother...

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Abstract

The invention discloses application of arglabin in the preparation of drugs for Stargardt disease, particularly type-I Stargardt disease. Arglabin plays the role of protecting not only retinal pigmentepithelia but also photoreceptor cells, and moreover, arglabin does not affect the functions of all the organs of the body and have no genetic toxicity, is highly safe, and is easy to administrate.

Description

technical field [0001] The invention relates to the technical field of medicines for Stargardt macular degeneration, in particular to the use of agaleb in the preparation of medicines for Stargardt macular degeneration. Background technique [0002] Stargardt macular degeneration is the most common hereditary macular degeneration of the retina, among which Stargardt disease type 1 (STGD1) is a type of early-onset autosomal recessive genetic eye disease caused by Abca4 gene mutation, which was first discovered in 1909 Reported by German ophthalmologist Karl Stargardt, its incidence rate is about 1 person in every 8000-10000 people, accounting for about 7% of macular degeneration diseases. More than 900 Abca4 mutations causing STGD1 have been reported so far, and the age of onset and severity of patients show great heterogeneity. Most patients with STGD1 develop the disease in juveniles or young adults, but some patients develop the disease after adulthood. It is generally be...

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Application Information

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IPC IPC(8): A61K31/365A61P27/02
CPCA61K31/365A61P27/02
Inventor 吴亚林廖怿
Owner XIAMEN UNIV
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