CRISPR-U™ technology (CRISPR based), developed by Ubigene, is more efficient than general
CRISPR/Cas9 technology in double-strand breaking and homologous recombination. With CRISPR-U™, Ubigene has
successfully edited over 3000 genes on more than 200 types of cell lines.
Objective
To create a Human NPC1 Knockout
model in cell line by CRISPR-U™-mediated genome engineering.
Target gene info
Official symbol
NPC1
Gene id
4864
Organism
Homo sapiens
Gene type
protein-coding
Official full symbol
NPC intracellular cholesterol transporter 1
Also known as
NPC, POGZ, SLC65A1
Genomic regions
Chromosome 18
Summary
This gene encodes a large protein that resides in the limiting membrane of endosomes and lysosomes and mediates intracellular cholesterol trafficking via binding of cholesterol to its N-terminal domain. It is predicted to have a cytoplasmic C-terminus, 13 transmembrane domains, and 3 large loops in the lumen of the endosome - the last loop being at the N-terminus. This protein transports low-density lipoproteins to late endosomal/lysosomal compartments where they are hydrolized and released as free cholesterol. Defects in this gene cause Niemann-Pick type C disease, a rare autosomal recessive neurodegenerative disorder characterized by over accumulation of cholesterol and glycosphingolipids in late endosomal/lysosomal compartments.
Strategy Summary
This gene has 0 protein coding transcripts：
Frame-shift
Fragment A
Fragment B
gRNA Detail
Strategy
Project Comprehensive Difficulty Assessment
According to the Red Cotton database: the CRISPR gene-editing strategy design is Unknown. Knockout project comprehensive difficulty is thus assessed as Unknown.
Red Cotton™ Notes
Gene
NPC1
had been KO in hepg2 cell line.
EZ-editor™ Gene Dependency
EZ-editor™ Gene Expression Level
EZ-editor™ Gene Copy Number
EZ-editor™ Gene Dependency
Result
The NPC1 gene you inquire is evaluated as high risk
in 1%
cell line.
Cell line is not selected, unable to assess the accurate risk level, for reference only.
In all cell lines, there is
0.1% cells with expression level below cutoff,
6.2% cells with low expression level,
93.8% cells with medium expression level
of NPC1 gene.
Cell line is not selected, unable to assess the accurate expression level, for reference only.
In all cell lines, there is
69.5% cells with low copy number,
26.3% cells with medium copy number,
4.2% cells with high copy number
of NPC1 gene.
Cell line is not selected, unable to assess the accurate copy number, for reference only.
Ubigene is an international high-technology enterprise focused on gene-editing cells. Our exclusive CRISPR-U™ technology has 10-20 times more efficient editing than traditional methods, easily achieving gene knockout, point mutation, and knock-in. Based on CRISPR-U™ technology, Ubigene has accumulated over 6000 successful gene-editing cases from more than 300 cell lines including iPSC and ESC, and has established a KO Cell Line Bank with 4500+ KO cell lines and Red Cotton™ gRNA Plasmid Bank with 10000+ gRNA plasmids available in stock.
Ubigene focuses on technological innovation and product development, of which EZ-editor™ series products that cover the whole workflow of gene-editing keep improving. Ubigene will move on toward our goal of "Make genome editing easier" and we won't stop!
EZ-editor™ Gene Dependency
EZ-editor™ Gene Expression Level 
EZ-editor™ Gene Copy Number 
