FFC#06/2015

Evaluation of the biological and therapeutic properties of mesoangioblasts-vessel associated progenitor cells in the cell based therapy of the cystic fibrosis disease

FFC#06/2015

Evaluation of the biological and therapeutic properties of mesoangioblasts-vessel associated progenitor cells in the cell based therapy of the 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

€ 60.000 €

RESULTS

Mouse MABs engrafted lung, tracheal and intestinal epithelium for up to 6 months in F508delCFTR mice after a single systemic injection. In vitro mMABS are able to express a functional CFTR channel and also in vivo mMABs transplanted KOCfrtm 1 UNC mice express a functional CFTR. Notably when transplanted and engrafted in the epithelium mMABs express typical epithelial markers, thus demonstrating mMAB ability to differentiate in epithelial cells. Finally, the first studies on human MABs demonstrated their ability to express a functional channel.

OTHER RESULTS

FFC #3/2024

Two molecules are effective in activating Heat Shock Proteins and enhancing the action of CFTR correctors with the F508del mutation in vitro.

FFC#5/2024

Some peptide nucleic acids (PNAs) re-sensitise Pseudomonas aeruginosa to the antibiotic meropenem in vitro and reduce its virulence.

FFC#1/2023

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