FFC#6/2016

Understanding the mode of action of regulatory pathways controlling F508del-CFTR proteostasis and developing drugs that rescue F508del-CFTR by targeting these pathways synergistically

AREA 1 Therapies to correct the underlying defect

FFC#6/2016

Understanding the mode of action of regulatory pathways controlling F508del-CFTR proteostasis and developing drugs that rescue F508del-CFTR by targeting these pathways synergistically
€ 0 still needed
0%
€ 100.000 goal

pRINCIPAL INVESTIGATOR

Alberto Luini (Consiglio Nazionale delle Ricerche, Dipartimento Scienze Biomediche, Istituto di Biochimica Proteica, Napoli)

Researchers

4

Category

AREA 1 Therapies to correct the underlying defect

Duration

2 years

Goal

€ 100.000

Funds raised

€ 100.000

Objectives

Researchers recently found cellular enzymes, termed proteostasis regulators and also efficient small molecule drugs that can inhibit these regulators so as to protect the mutant protein and restore the function of salt transport. The mechanism by which these regulators protect the mutant CFTR protein is not known and if uncovered if understood could an provide us with more specific drug targets. They will use state-of-the-art transcriptomic and proteomic methods to understand how these regulators act; then they will test small molecules inhibiting regulators in laboratory cell lines.

WHO ADOPTED THE PROJECT

Delegazione FFC di Cosenza Sud

€ 8.000

Delegazione FFC di Verbania e VCO

€ 12.000

Delegazione FFC di Bologna

€ 65.000

Delegazione FFC di Ferrara con il Gruppo di sostegno FFC di Comacchio

€ 15.000

Delegazione FFC di Verbania e VCO

€ 12.000

Delegazione FFC di Bologna

€ 65.000

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