General description
The induced pluripotent stem cell (iPSC) line derived from cystic fibrosis (CF) patients represents a significant breakthrough in CF research. This line incorporates the corrected version of the G542X mutation, a nonsense mutation in the CFTR gene that introduces a premature stop codon. This alteration results in the production of a truncated and nonfunctional cystic fibrosis transmembrane conductance regulator (CFTR) protein, disrupting chloride ion transport and leading to the characteristic thick mucus accumulation in the lungs and pancreas associated with cystic fibrosis.The iPSC line was generated using CRISPR-Cas9 technology, which utilizes a Cas9 nuclease to create precise double-strand breaks at the CFTR locus, guided by a CFTR-targeted guide RNA (gRNA). A repair single-stranded oligodeoxynucleotide (ssODN) serves as a template for genetic correction, effectively restoring functional CFTR protein expression.Providing both the CRISPR-corrected line and the line with the G542X mutation offers critical advantages for cystic fibrosis research. This dual approach facilitates direct comparative studies, enhancing our understanding of the molecular mechanisms underlying CF and clarifying the specific cellular dysfunctions caused by the G542X mutation. It also supports the development and validation of innovative therapeutic strategies, allowing the diseased line to serve as a platform for screening potential treatments while the corrected line verifies their efficacy.Overall, the availability of the CRISPR-corrected iPSC line, in conjunction with the diseased line, empowers researchers to advance cystic fibrosis research and promote the development of innovative treatment strategies that target the genetic basis of the disease.
Application
Each vial contains 6 viable cells.
Cells are tested negative for HPV-16, HPV-18, Hepatitis A, B, C, and HIV-1 & 2 viruses by PCR.
Cells are negative for mycoplasma contamination.
Each lot of cells is genotyped by STR analysis to verify the unique identity of the cell line.
Features and Benefits
Human induced pluripotent stem cell (iPSC) line derived from a cystic fibrosis (CF) patient with CRISPR-Cas9 corrrected G542X mutation.
Preparation Note
Human iPS cells should be stored in liquid nitrogen. The cells can be cultured for at least 10 passages after initial thawing without significantly affecting the cell marker expression and functionality.
Other Notes
This product is intended for sale and sold solely to academic institutions for internal academic research use per the terms of the “Academic Use Agreement” as detailed in the product documentation. For information regarding any other use, please contact
[email protected].
Disclaimer
Unless otherwise stated in our catalog or other company documentation accompanying the product(s), our products are intended for research use only and are not to be used for any other purpose, which includes but is not limited to, unauthorized commercial uses, in vitro diagnostic uses, ex vivo or in vivo therapeutic uses or any type of consumption or application to humans or animals.
