Fueled by advances in the field of genetics, the methods available to edit DNA sequences in living cells have continued to develop steadily. These technologies directly impact the fields of gene and cell therapy, where changes in the DNA sequence of target cells offer a route to correct genetic diseases and manipulate disorders like cancer. We review here the expanding menu of genome editing techniques and how they are being applied to therapeutic targets. The methods encompass a myriad of approaches to modify the covalent structure of DNA, including the targeted creation of double-strand breaks that can catalyze genomic changes, as well as the use of retroviruses and transposons to mediate gene addition, recombinases for sequence-specific gene addition and deletion, and base repair for direct sequence changes. The continued growth of the exciting field of genome editing is opening new possibilities for therapeutic intervention.
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http://dx.doi.org/10.1002/cpt.542 | DOI Listing |
Multiplexed assays of variant effect (MAVEs) perform simultaneous characterization of many variants. Prime editing has been recently adopted for introducing many variants in their native genomic contexts. However, robust protocols and standards are limited, preventing widespread uptake.
View Article and Find Full Text PDFAnim Reprod
January 2025
Faculdade de Zootecnia e Engenharia de Alimentos - FZEA, Universidade de São Paulo - USP, Pirassununga, SP, Brasil.
Somatic cell nuclear transfer (SCNT), or cloning, is used to reprogram cells and generate genetically identical embryos and animals. However, the cloning process is inefficient, limiting its application to producing valuable animals. In swine, cloning is mainly utilized to produce genetically modified animals.
View Article and Find Full Text PDFJ Community Hosp Intern Med Perspect
January 2025
University of Pennsylvania, Philadelphia, PA 19104, USA.
An examination of recent developments related to CRISPR technology, ethical considerations of the application of such technologies, and future directions for germline editing.
View Article and Find Full Text PDFFront Plant Sci
January 2025
Bio-resource Research and Utilization Joint Key Laboratory of Sichuan and Chongqing, Chongqing Institute of Medicinal Plant Cultivation, Nanchuan, Chongqing, China.
Introduction: Mitochondria are essential organelles that provide energy for plants. They are semi-autonomous, maternally inherited, and closely linked to cytoplasmic male sterility (CMS) in plants. , a widely used medicinal plant from the Caprifoliaceae family, is rich in chlorogenic acid (CGA) and its analogues, which are known for their antiviral and anticancer properties.
View Article and Find Full Text PDFOncol Res
January 2025
Institute of Biochemical Sciences, National Taiwan University, Taipei, 10617, Taiwan.
Background: Rho GTPases are essential regulators for cellular movement and intracellular membrane trafficking. Their enzymatic activities fluctuate between active GTP-bound and inactive GDP-bound states regulated by GTPase activating proteins (GAPs) and guanine nucleotide exchange factors (GEFs). Arhgap39/Vilse/Porf-2 is a newly identified GAP.
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