113. HSV Vector Development for Targeted Gene Delivery

design and development of a series of recombinant fusion vectors for targeted cancer gene delivery

Bioluminescence imaging after HSV amplicon vector delivery into brain.

BACKGROUND Firefly luciferase (Fluc) has routinely been used to quantitate and analyze gene expression in vitro by measuring the photons emitted after the addition of ATP and luciferin to a test sample. It is now possible to replace luminometer-based analysis of luciferase activity and measure luciferase activity delivered by viral vectors directly in live animals over time using digital imagin...

Trans-splicing ribozymes for targeted gene delivery.

Ribozymes are potential tools for genetic manipulation, and various naturally occurring catalytic RNAs have been dissected and used as the basis for the design of new endoribonuclease activities. While such cleaving ribozymes may work well in vitro, they have not proved to be routinely effective in depleting living cells of the chosen target RNA. Recently, trans-splicing ribozymes have been emp...

Targeted retrograde gene delivery for neuronal protection.

The cellular heterogeneity and complex circuitry of the central nervous system make it difficult to achieve precise delivery of experimental and therapeutic agents. We report here an in vivo retrograde gene delivery strategy to target mature projection neurons using adeno-associated virus, a vector with low toxicity and the capacity for long-term gene expression. Viral delivery to axon terminal...

Mannosylated liposomes for targeted gene delivery

BACKGROUND Liposomes can be modified with different ligands to control their biological properties, such as longevity, targeting ability, and intracellular penetration, in a desired fashion. The aim of this study was to modify liposomes with a novel mannosylated polyethylene glycol-phosphatidylethanolamine (M-PEG-PE) ligand to achieve active targeted gene delivery. METHODS Rat Kupffer cells w...