Despite the power of gene therapy, its successful application to medicine has been diminished due to: (i) high toxicities and potentially fatal adverse effects; (ii) poor transgene expression in target cells; and (iii) extensive vector degradation. While viral vectors greatly improve efficiency, they sometimes lead to cancers due to chromosomal integration and may suffer from a lack of desired tissue selectivity. In contrast, nonviral systems have proven safer, but less efficient. Developing highly effective and safe DNA vectors is essential.
The double-stranded DNA-minimal construct, called “DNA ministrings”, are miniaturized-DNA constructs carry only the expression cassette (promoter, gene of interest, intron, and nuclear translocation enhancing sequences). As such, DNA ministrings offer a much safer, yet highly effective alternative to current gene delivery vectors. TO BE CONT’D
Mediphage Bioceuticals Inc
Pharmacy / Pharmacology
University of Waterloo
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