Cell-based therapies have unique features for enhanced efficacy treatment against disease, targeted delivery, proliferation and differentiation into specific body locations. Cell therapy might require genetic engineering for the transfer of genetic material.
This genetic engineering is often achieved through transgene integration into the genome in a random manner which raises safety concerns since it may lead to instable gene expression, disruption of endogenous transcription and tumorigenicity.
The specific integration of genetic constructs into defined genetic safe harbor sites considerably enhances the safety and efficacy of the cell-based therapy. An ideal genetic safe harbor should not interfere with functional cellular genes, while it should allow for cell type-specific expression and active transcription protected from silencing mechanisms.
Therefore, identification of genomic safe harbor is challenging. NewBiologix exploits the full potential of gene transfer coupled to next-generation sequencing, combined with experimental validation to identify robust genomic safe harbors which are directly transferrable for cell therapy application.