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Trim72 as potential therapeutic target for als through ubiquitinating mutant fus protein

A mutant and ubiquitination technology, applied in the field of biomedicine, can solve the problems of unclear endogenous level expression and function of wild-type and mutant RBP

Pending Publication Date: 2020-02-04
神济昌华(北京)生物科技有限公司
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

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Problems solved by technology

Taken together, these data suggest that misprocessing of SG may contribute to disease etiology
However, it is unclear how endogenous levels of wild-type and mutant RBPs behave and function in SG formation and phase transitions, especially in disease-target neurons that are stress-challenged, partly due to the need for rational cell Urgent need for models and animal models

Method used

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  • Trim72 as potential therapeutic target for als through ubiquitinating mutant fus protein
  • Trim72 as potential therapeutic target for als through ubiquitinating mutant fus protein
  • Trim72 as potential therapeutic target for als through ubiquitinating mutant fus protein

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Embodiment

[0142] In the past, great efforts have been made to generate animal models for ALS by overexpressing recombinant DNA carrying mutants found in ALS families. Although these transgenic animal models have greatly increased our understanding of disease mechanisms, one could argue for potential artifacts resulting from ectopic overexpression of mutant proteins in disease models, such as in mice (a most popular ALS mouse 40-fold overexpression of human SOD1-G93A in model). Similar transgenic strategies were applied to generate TDP-43 models and FUS ALS models. However, overexpression of wild-type TDP-43 and FUS also produced locomotor phenotypes similar to mutant transgenes in many animal species, including Drosophila, mouse, and rat, suggesting a role for pathogenesis in these models There is an underlying human factor. In addition, transgenic strategies present other caveats, including uncertain genomic insertion sites, unstable copy number, potential disruption of genome integr...

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Abstract

Provided is a pharmaceutical composition comprising a scavenger, wherein the scavenger is used for clearing away mutant FUS. A method for treating or preventing or alleviating ALS is also provided.

Description

[0001] related application technical field [0002] Embodiments of the present disclosure generally relate to biomedicine, and more particularly to pharmaceutical compositions, uses of scavenging agents for clearing FUS mutants in the preparation of medicines, and methods for treating or preventing or alleviating ALS. Background technique [0003] Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease and motor neuron disease (MND), is a specific disease that causes the death of neurons that control voluntary muscles. Some also use the term "motor neuron disease" to refer to the group of conditions of which ALS is the most common. ALS is characterized by muscle stiffness, muscle twitching, and progressive weakness due to a decrease in muscle size. This causes difficulty speaking, swallowing and eventually breathing. In 90% to 95% of cases, the cause is unknown. About 5-10% of cases are inherited from an individual parent. About half of these inherited ca...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): A61K38/53A61K38/43A61P21/00
CPCA61P21/00A61K38/1709
Inventor 贾怡昌张雪
Owner 神济昌华(北京)生物科技有限公司
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