Cationic liposome mediated transgene expression in the guinea pig cochlea

Sensorineural hearing loss affects nearly 10% of the American population th at is refractory to conventional therapy. Gene therapy represents an interv ention with potential therapeutic efficacy. We studied the feasibility of c ationic liposome mediated gene transfer within the guinea pig cochlea in vi vo following direct microinjection into the cochlea. Transgene expression w as persistent up to 14 days in the neurosensory epithelia and surrounding t issue without toxicity and inflammation in the target organ. This study rep resents the first successful use of cationic liposomes for cochlear gene tr ansfer thus providing a safe and rapid alternative to the use of recombinan t viral vectors in gene therapy for inner ear disorders. (C) 1999 Elsevier Science B.V. All rights reserved.