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Htra1 modulation for treatment of AMD

A technology of regulatory regions and target sequences, applied in the fields of biology and medical applications, can solve problems such as no therapy

Pending Publication Date: 2022-01-21
UNIV OF UTAH RES FOUND
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

There are currently no approved therapies for early AMD or to prevent the development of AMD

Method used

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  • Htra1 modulation for treatment of AMD
  • Htra1 modulation for treatment of AMD
  • Htra1 modulation for treatment of AMD

Examples

Experimental program
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Embodiment Construction

[0064] The inventors have discovered that increasing HTRA1 mRNA and / or protein levels in the eye (eg, retinal pigment epithelium) provides a beneficial effect in subjects suffering from or at risk of developing age-related macular degeneration (AMD). In particular, increasing HTRA1 mRNA and / or protein levels provides a beneficial effect in patients suffering from the manifestations of chromosome 10-directed AMD (or "Chr 10AMD") or who are genetically predisposed to develop chromosome 10-directed AMD.

[0065] The present disclosure provides methods and agents for treating age-related macular degeneration (AMD), preventing its development, slowing its progression, reversing or ameliorating its symptoms and signs by increasing the expression or amount of HTRA1 in the eyes of a subject in need of treatment .

[0066] Methods of treating Chr 10AMD, preventing its development, slowing its progression, reversing or ameliorating its signs and symptoms are described. The method compr...

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Abstract

The invention provides compositions and methods for treatment of Chromosome 10-driven age-related macular degeneration, including gene therapy to increase HTRA1 expression in retinal pigmented epithelial cells in the eye.

Description

technical field [0001] The present invention relates to methods and compositions for treating age-related macular degeneration, as well as their applications in biological and medical fields. [0002] sequence list [0003] This application contains a Sequence Listing that has been filed electronically in ASCII format and is hereby incorporated by reference in its entirety. Said ASCII copy, formed on April 9, 2020, is named 098846-1185529-000810PC_SL.txt and is 46,642 bytes in size. [0004] Cross References to Related Applications [0005] This application claims the benefit of U.S. Provisional Application 62 / 832,182, filed April 10, 2019, which is hereby incorporated by reference in its entirety. Background technique [0006] Age-related macular degeneration (AMD) is the leading cause of irreversible vision loss in the developed world (for an overview see Zarbin, fur. Ophthalmol. 8:199-206, (1998)), affecting about 15% of people over the age of 60. An estimated 600 mil...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): C12N15/90C12N15/11C12N15/10C12N15/79C12N9/22
CPCC12N15/90C12N9/22C12N2310/20C12N15/63C12N2740/16043C12N2750/14143C12N2800/40C12N2830/008C07K2319/80C12N9/6424C12N15/1137C12Y304/21C12N2320/34C12N15/86A61P27/02A61K31/7088A61K48/0066C12N15/11
Inventor 布兰迪·威廉姆斯格雷戈里·哈格曼伯特·蒂莫西·理查兹奥萨马·约瑟夫
Owner UNIV OF UTAH RES FOUND
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