Review Article

The Trojan Horse Liposome Technology for Nonviral Gene Transfer across the Blood-Brain Barrier

Table 2

Tyrosine hydroxylase (TH) in brain and peripheral organs in the rat 3 days after intravenous injection of gene therapy with TH expression plasmids driven by either the SV40 promoter (clone 877) or the Gfap promoter (clone 951), respectively.

OrgansSaline (pmol/hr/mgp)TfRMAb-THL/877 (pmol/hr/mgp)TfRMAb-THL/951 (pmol/hr/mgp)

Ipsilateral striatum128 ± 27   5177 ± 446*   5536 ± 395*
Contralateral striatum6445 ± 5235832 ± 3915713 ± 577
Ipsilateral cortex176 ± 30132 ± 16184 ± 38
Contralateral cortex150 ± 36150 ± 24135 ± 25
Heart29 ± 345 ± 831 ± 3
Liver13 ± 2      130 ± 28**18 ± 6
Lung   42 ± 13    74 ± 2230 ± 6
Kidney24 ± 2 35 ± 531 ± 8

* difference from saline group (ANOVA with Bonferroni correction; rats per group). Rats were lesioned with intracerebral injections of 6-hydroxydopamine; 3 weeks after toxin injection, the rats were tested for apomorphine-induced rotation behavior; those rats testing positively to apomorphine were selected for gene therapy, which was administered intravenously 4 weeks after toxin administration; all animals were euthanized 3 days after gene administration. From [30].