FFC#5/2017

Dissecting the potency of human Mesoangioblasts to differentiate into CFTR-expressing epithelial cells: a step forward to an innovative cell-based therapy for Cystic Fibrosis disease

FFC#5/2017

Dissecting the potency of human Mesoangioblasts to differentiate into CFTR-expressing epithelial cells: a step forward to an innovative cell-based therapy for Cystic Fibrosis disease

PRINCIPAL INVESTIGATOR

Graziella Messina (Dipartimento di Bioscienze, Università degli Studi di Milano)

RESEARCHERS

7

CATEGORY

AREA 1 Therapies to correct the underlying defect

DURATION

1 year

GOAL

€ 30.000 €

RESULTS

Different populations of human MABs (hMABs) have been tested for the expression of CFTR. hMABS have been co-cultured with human bronchiolar epithelial cells from CF patients bearing severe mutations of CFTR to evaluate their ability to differentiate into epithelium and to express a functional CFTR (by Ussing chamber), thus mimicking the in vivo environment. Researchers observed that hMABs already in vitro express, although at low levels, both the immature and mature forms of CFTR. Notably, this expression corresponds, in ex vivo. Ussing chamber by co-cultures of hMABs with CF bronchiolar epithelial cells, to a functional CFTR channel. This project would represent a major advance over other cell-based therapeutic strategies for CF. This first study on human MABs demonstrates their ability to express functional CFTR, rescuing chloride ions transport in CF epithelia and possibly differentiating into epithelial-like cells. The results obtained from this proposal may make these cells eligible for clinical translation in human CF patients as a cell-based therapy.

OTHER RESULTS

FFC#1/2023

Tezacaftor, one of the components of Kaftrio, induces an accumulation of dihydroceramides both in vitro and in vivo in animal models

FFC#4/2023

Pseudopaline–aztreonam conjugates exhibited enhanced antimicrobial activity against Pseudomonas aeruginosa compared to aztreonam alone

FFC#10/2023

Several drugs already approved for human use inhibit the growth of P. aeruginosa, its virulence, or its ability to form biofilms in vitro