FFC#24/2014

The role of Glucocerebrosidase GBA2 in cystic fibrosis lung inflammation: from molecular mechanism to therapeutic strategies

FFC#24/2014

The role of Glucocerebrosidase GBA2 in cystic fibrosis lung inflammation: from molecular mechanism to therapeutic strategies

PRINCIPAL INVESTIGATOR

Sandro Sonnino (Dip. di Biochimica Medica e Medicina Traslazionale, Università di Milano)

RESEARCHERS

12

CATEGORY

AREA 4 Lung inflammation

DURATION

2 years

GOAL

€ 76.000

RESULTS

The data obtained in CF human epithelial bronchial cells (CFhEBC) support the role of GBA2 in the inflammatory response after Pseudomonas aeruginosa infection. An increased level of ceramide was responsible for the activation of the inflammatory response, which can be reduced through the inhibitory effect of a siRNA on GBA2 gene. So the researchers developed a lipid-based non-viral nanovector (NP) carrying that siRNA able to silencing GBA2 in vitro for up 8 days with any toxicity for the cells. The identification of GBA2, as well as others GSL-hydrolase as possible molecular targets related to the inflammatory response and development of innovative NP-based approach for their silencing, could represent a new therapeutic strategy in CF.

OTHER RESULTS

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