Hemoglobinopathies are a set of hereditary genetic illnesses caused by mutations in the hemoglobin beta (HBB) gene. The researchers used a combined supramolecular nanoparticle (SMNP)/supramolecular nano substrate-mediated delivery (SNSMD) approach to enable CRISPR-Cas9 knockin of the hemoglobin beta (HBB) gene into the adeno-associated virus integration site 1 (AAVS1) safe-harbor site of an engineered K562 3.21 cell line carrying the sickle cell disease mutation. This required the successive delivery of SMNP vectors harboring a Cas9•sgRNA combination and an HBB/green fluorescent protein (GFP)-encoding plasmid. Successful CRISPR-Cas9 knockin by HDR was performed, and further in vivo investigations confirmed the proliferative capability of HBB/GFP-knockin K562 3.21 cells after intraperitoneal injection into mice, laying the framework for broad gene therapy applications in hemoglobinopathies.
Figure 1. The researchers employed a combined SMNP/SNSMD approach to deliver EGFP-Cas9•sgRNA or HBB/GFP plasmid into K562 3.21 cells. (Yang P, et al., 2020)
Accelerate your gene editing research! Creative Biogene's Cas9 stable cell line K562 provides you with a stable and efficient platform for CRISPR-Cas9 gene editing experiments. This advanced cell line ensures continuous expression of Cas9, eliminating the need for repeated transfections, simplifying the experimental process, and improving the accuracy and reproducibility of gene editing. Whether it is used for gene modification of disease models or gene therapy research, Cas9 stable cell line-K562 is an indispensable tool for you. Accelerate your research progress, choose Creative Biogene's Cas9 stable cell line K562, and start your innovative gene editing journey!
Customer Q&As
How does the Cas9 Stable Cell Line-K562 ensure the continuous activity of the Cas9 enzyme?
A: The Cas9 Stable Cell Line-K562 ensures the continuous activity of the Cas9 enzyme by integrating the CRISPR-Cas9 system encoded by the Cas9 gene into the genome of the K562 cell line, utilizing specific promoters and selection markers. This integration allows the cells to continuously produce functional Cas9 enzyme without relying on external Cas9 expression vectors.
What is the role of the Cas9 Stable Cell Line-K562 in studying chronic myeloid leukemia (CML)?
A: The Cas9 Stable Cell Line-K562 provides a model for studying chronic myeloid leukemia (CML), particularly the function of the BCR-ABL fusion gene and targeted therapies. Through Cas9-mediated gene editing, scientists can simulate CML-associated gene mutations, leading to a better understanding of disease mechanisms and the development of new treatment strategies.
What is the application of the Cas9 Stable Cell Line-K562 in drug screening and development?
A: The application of the Cas9 Stable Cell Line-K562 in drug screening and development includes creating cell models with specific gene mutations to evaluate the therapeutic effects of potential drugs on CML cells. This cell line aids in accelerating the discovery and validation of new drugs, especially in the field of CML treatment.
What is the role of the Cas9 Stable Cell Line-K562 in studying gene expression regulation in CML cells?
A: The role of the Cas9 Stable Cell Line-K562 in studying gene expression regulation in CML cells is to investigate how the expression of specific genes affects the behavior of CML cells using Cas9-mediated gene editing technology. This helps reveal the molecular mechanisms of CML cells and provides information for developing new therapeutic targets.
What is the role of the Cas9 Stable Cell Line-K562 in studying drug resistance in CML cells?
A: The role of the Cas9 Stable Cell Line-K562 in studying drug resistance in CML cells is to simulate and investigate drug resistance-associated gene mutations through Cas9-mediated gene editing. This helps understand how CML cells develop resistance and how to develop strategies to overcome this resistance.
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Immediate gene editing
The Cas9 Stable Cell Line-K562, with Cas9 stably integrated, allows researchers to immediately proceed with CRISPR/Cas9 gene editing, eliminating time spent on constructing and validating expression systems, greatly accelerating the initiation of experiments.
Efficient gene knockout/knockin
Due to the stable expression of Cas9 protein in the Cas9 Stable Cell Line-K562, this cell line provides an efficient platform for gene knockout or knockin, making the study of gene functions and pathological mechanisms more efficient and accurate.
Optimized experimental conditions
The use of Cas9 Stable Cell Line-K562 simplifies the experimental workflow by eliminating the need for repeated verification of Cas9 protein expression, providing researchers with optimized and consistent experimental conditions, and reducing the risk of experimental failure.
Wide range of research applications
The Cas9 Stable Cell Line-K562 becomes an essential tool in hematology and cancer research due to its suitability for a variety of gene editing applications, including disease model construction, functional genomics studies, etc.
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