Identification of therapeutic candidates for Congenital Central hypoventilation Syndrome (CCHS)

Patent number:

EP23383165

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An in vitro screening method by NMR spectroscopy of potential therapeutic compounds for Congenital Central Hypoventilation Syndrome (CCHS). The method is based in the identification of active agents that inhibit secondary structure changes in PHOX2B transcription factor.

Countries:
Spain
Regions:
Community of Madrid
Centers:
CSIC
Other entities:
Sectors:
Health
Subsectors:
Pharmaceuticals
TRL Level:
TRL 3 – experimental proof of concept
BRL Level:
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Sustainable Development Goal:
Applications

Se han sintetizado varias construcciones de PHOX2B con diferentes regiones proteicas para mejorar la solubilidad y minimizar la complejidad espectral. Los cambios estructurales en PHOX2B expandido se monitorizan de forma sencilla, lo que permite un cribado de alto rendimiento de compuestos terapéuticos. La combinación de RMN y métodos de dispersión de luz proporciona una plataforma inigualable para identificar posibles compuestos terapéuticos.

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