Gene therapy with digital PCR – creating safe and effective therapies
Gene therapy has the potential to substantially impact treatment of monogenic disorders, and perhaps even lead to cures, by the transfer of genetic material into the appropriate cells of the body. Gene therapy may be performed using plasmids, but is often performed using viral-mediated gene transfer with Adeno-Associated Virus (AAV) or lentivirus.
AAV vectors are often preferred due to a smaller packaging size (~5 kb) that is useful for the delivery of small genes. Plus they are non-integrating and hence have relatively low systemic toxicity and cannot propagate without a helper virus, which makes them safe for clinical use.
Gene therapy workflow
Resources for gene therapy applications
Learn more about our products for gene therapy
Contamination control
Residual host-cell DNA (HCD) carryover poses major safety concerns when manufacturing protein and vaccine therapeutics. Risks include oncogenicity, infectivity and immunogenicity caused by your cell or gene therapy product. Therefore, levels of HCD must not exceed those established by regulatory agencies such as the U.S. Food and Drug Administration and the World Health Organization.
In comparison to qPCR, digital PCR provides higher sensitivity of detection at a lower template input range, enabling more robust applications. A multicopy target assay ensures that results are not affected by the fragmentation level of residual DNA (resDNA), a problem with qPCR.
Moreover, contaminating DNA in your PCR reagents can be a significant source of background and false positives. Ultra Clean Production enhances the specificity and efficiency of probe-based digital PCR to provide accurate, singleplex, or up to 5-plex analysis. Dedicated processes are implemented to enable Ultra Clean Production of the master mix, which minimizes contaminating DNA background and GC bias and gives you absolute certainty in your PCR reagents, making it an ideal choice for contamination-free, microbial applications as well as quality control applications, like residual DNA testing.
