Cell and Gene Therapy
TLA based targeted complete next generation sequencing of integrated vectors / transgenes, integration sites and of gene editing events empowers the development and quality control of gene and cell therapy products.
TLA analysis can be applied in different stages during development and application of gene therapy technology:
Characterization of the producer cell lines
- Identify the integration site(s);
- Study structural rearrangements in the host genome;
- Evaluate if targeting went correctly (e.g. no concatemerization, no off-target integrations);
- Characterize the vector/transgene sequence - SNPs and structural variants (e.g. deletions TG-TG fusions);
Evaluation of the transfected/transduced heterogenous cell samples
This means patient/donor-derived cellular gene therapy products, e.g. T-cells and B-cells:
- Identification of the integration sites (e.g. characterization of unique integration sites, preference of specific integration sites);
- Stability of the used vector (e.g. presence of SNPs and structural variants).
TLA is a very powerful tool to quality centrol genetically engineered cell lines used in the production of gene therapy products and to assess the genetic alterations resulting from the use of gene therapy products.
Webinar: Complete Genetic Characterisation of Transgenes, Integration Sites and Gene Editing Events in Gene Therapy Products