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New Findings from University of KwaZulu-Natal Yields New Data on Gene Therapy

February 6, 2014

By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Current study results on Biotechnology have been published. According to news originating from Durban, South Africa, by NewsRx correspondents, research stated, "Interest in hepatocyte-directed liposomal gene delivery is driven, in part, by the lack of effective treatment for several liver-associated disorders. To impart a hepatocyte targeting capability on DNA-lipoplexes, and to promote early release of cargo DNA from endosomes, novel glycosylated and imidazolylated cholesteryl derivatives have been synthesized and evaluated in vitro."

Our news journalists obtained a quote from the research from the University of KwaZulu-Natal, "Thus cholesteryl-3?-N-[(lactobionyl) amino] carbamate (Chol-LAC) and cholesteryl-3?-N-[(urocanyl) amino] carbamate (Chol-UAC) have been formulated with the cytofectin cholesteryl-3?-[(N',N'-dimethylaminopropyl) carbamate (Chol-T) and the neutral co-lipid dioleoylphosphatidyl ethanolamine (DOPE). Liposomes, which displayed a buffering capability at endosomal pH, effectively bound DNA at a N/P ratio of 0.8:1 and offered partial protection against serum nuclease digestion. The MTT cell viability assay showed that lipoplexes were well tolerated by human hepatoma cells (HepG2), which were efficiently transfected almost exclusively by asialoglycoprotein receptor (ASGP-R)-mediation, as demonstrated in competition assays. In the ASGP-R-negative human kidney cell line (HEK293) transfection levels were considerably lower (p

According to the news editors, the research concluded: "Therefore the combination of Chol-LAC and Chol-UAC in cationic liposomal formulations may provide a platform for the development of useful hepatotropic gene delivery systems."

For more information on this research see: Glycosylated liposomes with proton sponge capacity: novel hepatocyte- specific gene carriers. Current Drug Delivery, 2013;10(6):685-95. (Bentham Science Publishers -; Current Drug Delivery -

The news correspondents report that additional information may be obtained from S. Habib, Discipline of Biochemistry, University of KwaZulu-Natal, P Bag X54001, Durban 4000, South Africa. Additional authors for this research include M. Singh and M. Ariatti (see also Biotechnology).

Keywords for this news article include: Biotechnology, Durban, Carbamates, Hepatocytes, South Africa, Gene Therapy, Acyclic Acids, Bioengineering, Gastroenterology.

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Source: Gene Therapy Weekly

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