Bifunctional compounds for targeted hepatic gene delivery
نویسندگان
چکیده
منابع مشابه
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...
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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...
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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...
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ژورنال
عنوان ژورنال: Gene Therapy
سال: 2007
ISSN: 0969-7128,1476-5462
DOI: 10.1038/sj.gt.3302917