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Content archived on 2024-05-24

Evolving evidence based treatment strategies for infantile hyperinsulinism using clinical, genetic and cell biological insights into a heterogenous disease

Objective

Infantile hyperinsulinism (HI) is a rare devastating heterogeneous genetic disease. Germ line or somatic mutations in one of four genes (resulting in loss / reduction of ATP dependent potassium currents in the pancreatic b-cell membrane) are founding 50 % of individuals. Overall 35% of patients have a somatic mutation within an abnormal focus of pancreas ( foHI ). The genetic basis in other patients is obscure. This proposal will
(a) screen new candidate genes identified through study of clinical phenotype and b-cell biology in vitro;
b) evolve strategies to identify foHI early and facilitate early specialist surgery in supra-regional surgical centres;
(c) critically evaluate / develop new treatments for non-foHI;
(d)establish a multinational patient / genetic database;
(e) establish and cryopreserve HI b-cell lines for future genetic manipulation and transplantation studies.

Fields of science (EuroSciVoc)

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Topic(s)

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Call for proposal

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Funding Scheme

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CON - Coordination of research actions

Coordinator

UNIVERSITY COLLEGE LONDON
EU contribution
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Address
30,Guilford Street 30
WC1N 1EH LONDON
United Kingdom

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Total cost

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Participants (12)

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