Acta Pharm. 48 (1998) 221-227

Review article  
 

Current status of cationic liposome-mediated gene therapy in cystic fibrosis

ALLESIA COLOSIMO,1  FEDERICA SANGIUOLO,1 SABRINA DI SARIO,2
PAOLA AMICUCCI,1 ANNA LUCIA SERAFINO,2  ANTONIO NOVELLI,1
MARGHERITA SABANI,1  MASSIMO CASTRO,3 VINCENZINA LUCIDI,3
GIUSEPPE MOSSA,2 BRUNO DALLAPICCOLA,4  and GIUSEPPE NOVELLI1

1Cattedra di Genetica Umana e Medica, Dipartimento di Biopatologia e Diagnostica Per Immagini, Università di Roma »Tor Vergata« Rome, Italy   2Istituto di Medicina Sperimentale C.N.R. Rome, Italy  3Divisione di Pediatria Ospedale Bambino Gesu', Rome, Italy  4Ospedale CSS, San Giovanni Rotondo Rome, Italy 
Received September 16, 1998      Accepted November 23, 1998

Gene therapy is actually seen as a useful approach to the treatment of a wide range of diseases, including single-gene disorders and polygenic diseases of multifactorial ethiology. Cystic fibrosis (CF), one of the most common single-gene diseases, has become a main target for treatments by somatic gene therapy. Human clinical trials have been performed in the past five years, using cationic liposome and adenovirus as vectors. The cationic liposome approach and the current progress in CF gene therapy are here briefly reviewed.


Keywords: cystic fibrosis, gene therapy, cationic liposomes, human clinical trials