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 C9orf72 Knockout
model in cell line by CRISPR-U™-mediated genome engineering.
Target gene info
Official symbol
C9orf72
Gene id
203228
Organism
Homo sapiens
Gene type
protein-coding
Official full symbol
C9orf72-SMCR8 complex subunit
Also known as
ALSFTD, DENND9, DENNL72, FTDALS, FTDALS1
Genomic regions
Chromosome 9
Summary
The protein encoded by this gene plays an important role in the regulation of endosomal trafficking, and has been shown to interact with Rab proteins that are involved in autophagy and endocytic transport. Expansion of a GGGGCC repeat from 2-22 copies to 700-1600 copies in the intronic sequence between alternate 5' exons in transcripts from this gene is associated with 9p-linked ALS (amyotrophic lateral sclerosis) and FTD (frontotemporal dementia) (PMID: 21944778, 21944779). Studies suggest that hexanucleotide expansions could result in the selective stabilization of repeat-containing pre-mRNA, and the accumulation of insoluble dipeptide repeat protein aggregates that could be pathogenic in FTD-ALS patients (PMID: 23393093). Alternative splicing results in multiple transcript variants encoding different isoforms.
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
C9orf72
had been KO in hek293t cell line.
EZ-editor™ Gene Dependency
EZ-editor™ Gene Expression Level
EZ-editor™ Gene Copy Number
EZ-editor™ Gene Dependency
Result
The C9orf72 gene you inquire is evaluated as high risk
in 0%
cell line.
Cell line is not selected, unable to assess the accurate risk level, for reference only.
In all cell lines, there is
2.6% cells with expression level below cutoff,
83.7% cells with low expression level,
13.6% cells with medium expression level
of C9orf72 gene.
Cell line is not selected, unable to assess the accurate expression level, for reference only.
In all cell lines, there is
76.2% cells with low copy number,
20.1% cells with medium copy number,
3.7% cells with high copy number
of C9orf72 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 
