Giovanna Sersale

TL;DR: A nonviral approach for transfecting various airway-derived cell lines in vitro showed that cationic polymers and lipids are able to transfect with high efficiency the reporter genes firefly luciferase and E. coli lacZ.

TL;DR: Cardiac involvement was present early in more than a half of the patients identified as having mucopolysaccharidosis, and generally progressed, being more frequent and severe in the first and second types of the disease.

TL;DR: It is concluded that a salt-losing syndrome with tubular resistance to aldosterone can occur during pyelonephritis in young infants with congenital UT malformation, that the risk diminishes considerably or disappears after 3 months of age and that in the absence ofUT malformation pyel onephritis does not cause acute sodium loss of clinical relevance.

TL;DR: In this study, a group of MPS IIIC patients, who are mainly of Italian origin, have been clinically characterized and mutational analysis of the HGSNAT gene resulted in the identification of nine alleles, of which eight are novel.

TL;DR: The results shown in this study might be helpful in considering cationic vectors as therapeutic nonviral vectors for transferring CFTR into human CF respiratory cells, as well as for restoring the bacterial killing activity defective in cystic fibrosis.