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TKI type switching overcomes ROS1 L2086F in ROS1 fusion-positive cancers.
NPJ Precis Oncol
August 2024
Department of Pediatrics, Oregon Health & Science University, Portland, OR, 97239, USA.
Article Synopsis
- ROS1 tyrosine kinase inhibitors (TKIs) are effective for treating ROS1-positive non-small cell lung cancer, but the emergence of resistance mutations, particularly L2086F, poses a challenge.
- The study compares the efficacy of different TKIs, revealing that type II TKIs maintain effectiveness against the L2086F mutation while type I TKIs do not, highlighting important structural differences in their binding.
- Results indicate that cabozantinib is effective against ROS1 L2086F in clinical cases, but its multi-kinase activity suggests the need for more targeted options, with gilteritinib offering potential benefits worth further exploration.
Whole genome sequencing of CRISPR-Cas9-edited Mauritian cynomolgus macaque blastomeres reveals large-scale deletions and off-target edits.
Front Genome Ed
January 2023
Wisconsin National Primate Research Center, University of Wisconsin-Madison, Madison, WI, United States.
Genome editing by CRISPR-Cas9 approaches offers promise for introducing or correcting disease-associated mutations for research and clinical applications. Nonhuman primates are physiologically closer to humans than other laboratory animal models, providing ideal candidates for introducing human disease-associated mutations to develop models of human disease. The incidence of large chromosomal anomalies in CRISPR-Cas9-edited human embryos and cells warrants comprehensive genotypic investigation of editing outcomes in primate embryos.
View Article and Find Full Text PDFTherapeutic correction of hemophilia A using 2D endothelial cells and multicellular 3D organoids derived from CRISPR/Cas9-engineered patient iPSCs.
Biomaterials
April 2022
Laboratory of Stem Cells and Tissue Regeneration, Department of Biotechnology, College of Life Sciences and Biotechnology, Korea University, Seoul, 02841, South Korea. Electronic address:
The bleeding disorder hemophilia A (HA) is caused by a single-gene (F8) defect and its clinical symptom can be substantially improved by a small increase in the plasma coagulation factor VIII (FVIII) level. In this study, we used F8-defective human induced pluripotent stem cells from an HA patient (F8d-HA hiPSCs) and F8-corrected (F8c) HA hiPSCs produced by CRISPR/Cas9 genome engineering of F8d-HA hiPSCs. We obtained a highly enriched population of CD157 cells from CRISPR/Cas9-edited F8c-HA hiPSCs.
View Article and Find Full Text PDFFunctional analysis of TaPDI genes on storage protein accumulation by CRISPR/Cas9 edited wheat mutants.
Int J Biol Macromol
January 2022
Institute of Crop Sciences, Chinese Academy of Agricultural Sciences, 100081 Beijing, China. Electronic address:
Wheat protein disulfide isomerase (PDI) is involved in the formation of glutenin macropolymers (GMP) and the correct folding and accumulation of storage proteins in endosperm. In present study, seven types of homozygous TaPDI gene edited mutants were obtained by CRISPR/Cas9 technology, which were confirmed by PCR-RE and sequencing. Compared with other mutants and wild type (WT), the grain length and width in mutant PDI-abd-6 which was edited for the three TaPDI homoeologous genes were reduced, and the grain middle parts were slumped.
View Article and Find Full Text PDFTherapeutic Genome Editing and In Vivo Delivery.
AAPS J
June 2021
Department of Pharmaceutical and Biomedical Sciences, College of Pharmacy, University of Georgia, Athens, Georgia, 30602, USA.
Improvements in the understanding of human genetics and its roles in disease development and prevention have led to an increased interest in therapeutic genome editing via the use of engineered nucleases. Various approaches have been explored in the past focusing on the development of an effective and safe system for sequence-specific editing. Compared to earlier nucleases such as zinc finger nuclease and transcription activator-like effector nuclease, the relatively low cost and ease of producing clustered regularly interspaced short palindromic repeats associated protein 9 (CRISPR/Cas9) systems have made therapeutic genome editing significantly more feasible.
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