Gene-Modulation Technologies in the Development of Cell-Based Therapies

Cell therapy, especially adoptive T-cell transfer, is an active area of cancer research and treatment. Although promising, many challenges from efficacy to safety remain.

Horizon Discovery harnesses various gene-modulation technologies, such as CRISPR, rAAV, ZFN, siRNA, and shRNA, to successfully engineer primary cells and cancer cell lines. Whole-genome pooled and targeted arrayed functional genomic screens are performed that extend to primary human T cells. Such screens could be used to identify new targets that improve CAR T-cell survival in the immunosuppressive tumor microenvironment, increase migration and infiltration, or reduce immunogenicity. Potential targets identified from these screens may be validated with follow-up in vitro or in vivo studies. In this webinar, current data from primary immune cells will illustrate how CRISPR–Cas9 and RNAi can extend the use of adoptive T-cell transfer in the clinic.


Topics to be covered:

  • Overview of gene-modulation technologies and functional genomic screens
  •  Areas in which CRISPR-Cas9 and RNAi could be used to extend the use of adoptive T-cell transfer in the clinic

To learn more about information-rich solutions for CAR-T research from Sartorius, visit


John O'Rourke, PhD
Head of Product Development, Cell Analytics

Verena Brucklacher-Waldert, PhD
Principal Scientist
Horizon Discovery Ltd.

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