FFC#5/2015

The plant cytokine kinetin and its analogues as potential therapeutic agents to correct CFTR splicing defects

AREA 1 Therapies to correct the underlying defect

FFC#5/2015

The plant cytokine kinetin and its analogues as potential therapeutic agents to correct CFTR splicing defects
€ 0 still needed
0%
€ 60.000 goal

pRINCIPAL INVESTIGATOR

Stefano Duga (Università Humanitas, Milano)

Partner

Lucy Costantino (UOS Lab. di Genetica Medica – Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, Milano); Christian Orrenius (Computational Sciences Chemical Core Technologies Department – Nerviano Medical Sciences Srl, Nerviano, Milano)

Researchers

11

Category

AREA 1 Therapies to correct the underlying defect

Duration

2 years

Goal

€ 60.000

Funds raised

€ 60.000

Objectives

Recent advances in CF treatment aim to develop specific drugs targeting classes of molecular defects underlying CF. No such therapies are available for patients carrying splicing mutations. About 13% of CF mutations are classified as splicing defects. The plant hormone kinetin was shown to correct RNA aberrant splicing in 2 severe genetic diseases (familial dysautonomia and neurofibromatosis). This project aims to investigate the therapeutic potential of kinetin to treat CF splicing defects and to perform a search of other molecules similar to kinetin. The ability of kinetin to normalize splicing in different cell model systems and subsequently in cell lines derived from patients carrying splicing defects will be tested. In parallel, novel compounds similar to kinetin by a computational screening of a virtual library will be searched.

WHO ADOPTED THE PROJECT

Delegazione FFC di Roma

€ 60.000

Delegazione FFC di Belluno con i Rocciatori di Fonzaso

€ 25.000

Delegazione FFC di Tradate Gallarate

€ 20.000

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