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Diagnostic and therapeutic methods

A technology of amyloidosis and active agents, applied in the field of diagnostic methods and kits, pharmaceutical compositions for treatment or prevention, and able to solve problems such as the unclear role of HS

Active Publication Date: 2014-01-01
李晋萍
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

Although accumulating evidence suggests that HS plays an important role in amyloid deposition, the exact role of HS in the pathology of amyloidosis remains unclear

Method used

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  • Diagnostic and therapeutic methods
  • Diagnostic and therapeutic methods
  • Diagnostic and therapeutic methods

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Embodiment

[0069] It is obvious to a person skilled in the art that, as technology advances, the inventive concept can be implemented in various ways. The invention and its embodiments are not limited to the examples described below but vary within the scope of the claims.

[0070] Materials and methods

[0071] Aggregation of TTR

[0072] Recombinant wild-type TTR was expressed in bacteria and purified as described above (Furuya et al. (1991) Biochemistry 30:2415-2421). Purified proteins were dissolved in 20 mM phosphate-citrate buffer (pH 2.7, 3.5, 4.5 or 7.4) to a final concentration of 50 μM (for Thioflavin T fluorescence) or 500 μM (for non-denaturing PAGE). Heparin (3H-labeled or unlabeled) and HS were added to the TTR solution to the concentrations indicated by the reference symbols. Samples were incubated at 37°C for the stated times.

[0073] TTR peptide

[0074] Synthetic peptides corresponding to different TTR fragments were purchased from commercial sources or synthesize...

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Abstract

The present invention relates to a method of treating or preventing transthyretin amyloidosis, pharmaceutical composition for use in said treatment or prevention, as well as to a diagnostic method and a kit.

Description

field of invention [0001] The present invention relates to a method for treating or preventing transthyretin amyloidosis, a pharmaceutical composition for the treatment or prevention, a diagnostic method and a kit. Background of the invention [0002] Transthyretin (TTR) is a functional plasma protein composed of four 14 kDa subunits. TTR is mainly synthesized by the liver and functions as a transporter of retinol and thyroxine. In addition to its physiological function, TTR has been found to be a major component of amyloid fibrils in several different clinical forms of amyloidosis, including familial amyloid polyneuropathy (FAP), familial cardiac amyloidosis, and sporadic Senile systemic amyloidosis (SSA). SSA is caused by selective deposition of wild-type (WT) TTR fibrils in cardiac tissue and affects almost 25% of the population over the age of 80. [0003] About 100 amino acid mutations of TTR have been associated with familial forms of TTR amyloidosis. Studies have ...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): G01N33/68
CPCG01N2800/28G01N2800/32G01N2800/7047G01N33/6893G01N33/6896A61P43/00A61P7/02A61P9/00A61K31/727G01N2333/47
Inventor U·林达尔J·李F·诺博恩
Owner 李晋萍
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