Insights into the influences of carboxymethyl-β-cyclodextrin on DNA formulations characteristics and gene transfection efficiency

Elsana, Hassan, Mysina, Svetlana, Elkordy, Emal Ali, Carr-Wilkinson, Jane and Elkordy, Amal (2018) Insights into the influences of carboxymethyl-β-cyclodextrin on DNA formulations characteristics and gene transfection efficiency. Current Drug Delivery, 15 (6). pp. 867-878. ISSN 1567 2018

Document

Author accepted version Elkordy.pdf - Accepted Version

Abstract

Official URL: https://doi.org/10.2174/1567201815666180226115503

Abstract

BACKGROUND

Gene therapy is an expanding field and it can treat genetic and acquired diseases.

OBJECTIVE

It was found that formulations with DNA: CM-β-CD (Carboxymethyl-beta-cyclodextrin): Pluronic-F127 1:3:3 and 1:3 DNA: CM-β-CD are the most stable formulations indicating high incorporation of DNA within CM-β -CD.

METHOD

Gel electrophoresis revealed DNA with low CM-β -CD concentration has formed a more stable complex. Samples 1:3 DNA: CM-β-CD and 1:3:3 DNA: CM-β-CD: Pluronic-127 show no DNA fragment, suggesting good condensation of DNA inside cyclodextrin cavity.

RESULTS

This was confirmed by fluorescence data where fluorescence intensity was reduced for samples DNA: CM-β-CD 1:3. Overall, the findings showed that Carboxymethyl-beta-cyclodextrin (as a novel non-viral gene vector) was able to provide condensation and protection to the DNA, with and without Pluronic-F127, at low concentration.

CONCLUSION

pDNA/CM-β-CD complex has not only shown to be able to transfect COS 7 and SHSY5Y cell lines, but it gives a higher transfection efficiency than that produced by the TransIT-LT1 commercial transfection reagent.