FFC#1/2020

Peptide-nucleic acids as potential CFTR amplifier molecules for cystic fibrosis treatment

AREA 1 Therapies to correct the underlying defect

FFC#1/2020

Peptide-nucleic acids as potential CFTR amplifier molecules for cystic fibrosis treatment
€ 0 still needed
0%
€ 85.000 goal

pRINCIPAL INVESTIGATOR

Felice Amato (Centro CEINGE – Biotecnologie Avanzate, Napoli, Lab. di ricerca in fibrosi cistica)

Researchers

8

Category

AREA 1 Therapies to correct the underlying defect

Duration

2 years

Goal

€ 85.000

Funds raised

€ 85.000

Objectives

The project intends to improve the positive effects of CFTR modulators, by increasing CFTR protein synthesis and thus providing abundant substrate to correctors and potentiators for the greatest possible protein’s recovery and normal CFTR function. The candidate molecules are peptide nucleic acids (PNA); they will be tested in primary respiratory cells from CF patients, carrying residual function mutations (RF). The PNA can both inhibit and amplify the transcription of the genetic code from gene DNA to messenger RNA, which is the mediator for the synthesis of the CFTR protein. Several PNAs will be produced and tested, selecting those with greater amplification activity, suitable as drug candidates, which could be further tested in humans in association with the available CFTR modulators.

who adopted the project

Delegazione FFC di Bologna

€ 85.000

Delegazione FFC Ricerca di Firenze

€ 45.000

Delegazione FFC Ricerca di Ascoli Piceno

€ 12.950

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FFC#2/2025

Developing RNA-targeted therapies to regulate gene expression and restore CFTR synthesis