TREATMENTS (NON-ANTIEPILEPTIC DRUGS) | Immunoliposome Gene Therapy and Its Potential Use in Treatment of the Epilepsies

Despite advances during the decade of the brain, there is still no effective treatment for neurodegenerative disorders and many monogenic diseases. The perception that delivery across the blood–brain barrier (BBB) is an insurmountable problem and deaths reported in gene therapy using viral vectors have led to the development of pegylated immunoliposomes (PILs) as nanocantainer (85 nm) vehicles for delivering the replacement gene to the brain. Release of the liposome cargo has been successfully demonstrated within CNS or astrocytoma of mice, when RNAi targets EGFR in SCID mice, and when tyrosine hydoxylase is delivered to brain in murine parkinsonism. Now, brain delivery of the Laforin gene using PILs is showing great promise in fatal Lafora epilepsy.