Importance Of Data Reproducibility For Development Of Safe Gene Therapies

Viral-based vectors have now become an integral part of clinically approved gene therapies. Ongoing optimization of AAV vector efficiency and specificity, as well as the discovery of new adenovirus strains in nature, offer opportunities for new AAVs to be explored as potential therapeutic constructs. New vector strains and designs have the potential to introduce genetic elements that elicit an immune response, so they must be investigated for both efficacy and safety. When the therapeutic product, viral capsid, or other vector elements may be culprits in gene therapy-induced toxicity, exploring activation or silencing of gene expression may help to guide vector design and construction to balance therapeutic efficacy against potential toxicity.

Follow along with an introduction to the importance of reproducible quantitation of gene expression to investigate potential sources of vector-induced immunotoxicity in gene therapy, along with a demonstration of well-to-well and instrument-to-instrument data concordance across multiple biosystems.