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Opendata, web and dolomites

GHSO SIGNED

Generation of human steroid-producing organoids: a new approach to treat adrenal insufficiency

Total Cost €

EC-Contrib. €

Partnership

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GHSO project word cloud

Explore the words cloud of the GHSO project. It provides you a very rough idea of what is the project "GHSO" about.

temporally utilize reversible healthy lacking synthesis efficient translation life requiring volume cellular pattern advantages exogenous functional hormonal stress organoids homeostasis cells overexpression paradigm cortisol mammalian individuals human found models drug platform source genome transform therapy tractable metabolism steroids site cah generate crispr salt suffering steroid bench cell mineralocorticoids engineered insufficiency small hescs directed glucocorticoids hyperplasia molecules disease adrenal quality lifelong mutations stem vivo variables congenital accelerate diurnal cas9 mediate protein faster offers transcription carbohydrate suitably replacement primary pluripotent disorders blood cortex harbouring patients ai tunable threatening functions therapies mimics reprogramming specialized embryonic noted producing

Project "GHSO" data sheet

The following table provides information about the project.

Coordinator	TECHNISCHE UNIVERSITAET DRESDEN Organization address address: HELMHOLTZSTRASSE 10 city: DRESDEN postcode: 1069 website: http://www.tu-dresden.de/ contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.
Coordinator Country	Germany [DE]
Total cost	246˙669 €
EC max contribution	246˙669 € (100%)
Programme	1. H2020-EU.1.3.2. (Nurturing excellence by means of cross-border and cross-sector mobility)
Code Call	H2020-MSCA-IF-2018
Funding Scheme	MSCA-IF-GF
Starting year	2019
Duration (year-month-day)	from 2019-06-01 to 2022-05-31

Partnership

Take a look of project's partnership.

#	participants	country	role	EC contrib. [€]
1	TECHNISCHE UNIVERSITAET DRESDEN TECHNISCHE UNIVERSITAET DRESDEN Organization address address: HELMHOLTZSTRASSE 10 city: DRESDEN postcode: 1069 website: http://www.tu-dresden.de/ contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	DE (DRESDEN)	coordinator	246˙669.00
2	CHILDREN'S HOSPITAL CORPORATION CHILDREN'S HOSPITAL CORPORATION Organization address address: LONGWOOD AVENUE 300 city: BOSTON postcode: 2115 website: http://www.childrenshospital.org/ contact info title: n.a. name: n.a. surname: n.a. function: n.a. email: n.a. telephone: n.a. fax: n.a.	US (BOSTON)	partner	0.00

Map

Project objective

The adrenal cortex is essential for life; it is the primary site of steroid synthesis, producing glucocorticoids, which affect carbohydrate metabolism and mediate the mammalian stress response and mineralocorticoids, which control blood volume and salt homeostasis. Adrenal insufficiency (AI), which can be life threatening, is cause by a number of adrenal disorders, and lifelong management of these patients with exogenous steroids can be challenging. No drug suitably mimics the diurnal pattern of cortisol noted in healthy individuals, and objective variables to measure hormonal replacement therapy quality are lacking. The ability to generate steroid-producing cells from pluripotent stem cells through cell reprogramming, a process where a specialized cell type is induced to transform into a different cell, offers a new paradigm for functional studies, modelling human disease and drug testing and eventually can be used as a cell source for cellular therapies for patients suffering from adrenal conditions. This proposal aims to develop methodologies to generate adrenal-like organoids from human embryonic stem cells (hESCs), which have not been generated so far, without requiring overexpression of exogenous transcription factors and test them in in vivo models of adrenal insufficiency. Because small molecules provide several distinct advantages in controlling protein functions (e.g., temporally controllable, reversible, tunable and tractable) I will utilize them for a faster, more efficient, and directed cellular reprogramming. CRISPR-Cas9 genome engineered steroid-producing organoids harbouring common mutations found in congenital adrenal hyperplasia (CAH), the most common type of AI, will be generated and used as a disease modelling platform to study CAH. This proposal aims to accelerate the translation of this promising bench research to patients affected by adrenal insufficiency over the next 10 years.

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The information about "GHSO" are provided by the European Opendata Portal: CORDIS opendata.