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The Application Progress and Prospects of Gene Editing Technology in the Field of Biopharmaceuticals

Author(s)

Junhao Yang*

Affiliation(s)

Macau University of Science and Technology, Macau, China *Corresponding Author.

Abstract

This study systematically reviews the application progress of gene editing technology in the field of biopharmaceuticals, analyzes the technology development trend and industrialization prospects. Clustered Regularly Interspaced Short Palindromic Repeats and CRISPR-associated protein 9 (CRISPR-CAS9) technology has made breakthrough progress in the field of biopharmaceuticals with its precise positioning of guide Ribonucleic Acid (RNA) and efficient cleavage ability of CAS9 protein. In cell engineering, the efficiency of recombinant protein production has been significantly improved through metabolic pathway optimization, anti-apoptosis mechanism enhancement and precise regulation of glycosylation. In the field of cell therapy, gene editing technology has achieved optimization of Chimeric Antigen Receptor T-Cell (CAR-T cell) therapy, and the universal Universal Chimeric Antigen Receptor T-cell 19 (UCART19) have a complete remission rate of 82% for relapsed/refractory B-cell leukemia. In drug development, the construction of precise disease models has increased the efficiency of lead compound screening by 5-10 times. CRISPR therapy has restored hemoglobin levels to 80% of the normal range in the treatment of β-thalassemia. Gene editing technology is driving biopharmaceuticals into a new era of precision medicine, but it still needs to be continuously improved in terms of off-target effect control, delivery system optimization and safety assessment.

Keywords

Gene Editing, CRISPR-CAS9, Biopharmaceuticals, Cell Engineering, Molecular Biology

References

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