Work

CRISPR-Edited Immune Effectors: The End of the Beginning

Year: 2020

Type: article

Abstract: Genome editing has emerged as a powerful tool to enhance the potency of effector cell therapies and potentially improve their safety.1 Stadtmauer et al.2 at the University of Pennsylvania recently reported preliminary results of a first-in-man clinical study using CRISPR/Cas9-edited therapeutic T cells in patients with advanced malignancies. In this study, peripheral T cells were obtained from patients with multiple myeloma or sarcoma. To allow their engineered T cells to be more resistant to tumor-mediated immune suppression, the cells were then activated and treated with a mixture of CRISPR/Cas9 ribonucleoproteins (RNPs) to disrupt the PDCD1 gene that encodes inhibitory receptor PD-1 as well as TRAC and TRBC genes.

Source: Molecular Therapy

DOI: 10.1016/j.ymthe.2020.03.009

Authors: Feiyan Mo, Helen E. Heslop, Maksim Mamonkin

Institutions: Houston Methodist, Baylor College of Medicine, Texas Children's Hospital

Language: English

Topic: CAR-T cell therapy research

Subfield: Oncology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: hybrid

Estimated APC paid: $3,500