Abstract
Genetically engineered T-cell therapy holds great potential for the curative treatment across a series of cancers. However, drug-related safety concerns need to be addressed in the emerging medicine of the future
Highlights
Despite the variety of traditional methods available to treat cancer, such as surgical resection, chemotherapy, and radiation therapy, immunotherapy has emerged as an attractive alternative for cancer patients
chimeric antigen receptor (CAR)-T cells are modified with a chimeric receptor molecule, composed of an extracellular antigenbinding domain, a hinge, a transmembrane domain, and intracellular domain(s), to recognize antigens on cell surfaces independent of the major histocompatibility complex (MHC) [2]
To maximize the transduction efficiency, AAV are used for delivery of homology directed repair (HDR) templates containing a gene expression cassette like CAR or T cell receptor (TCR), while CRISPR endonuclease is usually electroporated in the form of mRNA or ribonucleoprotein (RNP)
Summary
Despite the variety of traditional methods available to treat cancer, such as surgical resection, chemotherapy, and radiation therapy, immunotherapy has emerged as an attractive alternative for cancer patients. The optimization of the CRISPR-Cas9 genome-targeting system enables the precise knock-in of large DNA fragments in the genomes of the human primary T cell, allowing for more therapeutic gene modifications. By co-electroporation of Cas9 RNP and long dsDNA templates, Roth [13] successfully corrected the c.530A>G IL2RA mutation in the T cells from three compound heterozygous siblings, which rescued IL-2Rα cell surface expression on CD3+ T cells two days after treatment.
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