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 HTT Knockout
model in cell line by CRISPR-U™-mediated genome engineering.
Target gene info
Official symbol
HTT
Gene id
3064
Organism
Homo sapiens
Gene type
protein-coding
Official full symbol
huntingtin
Also known as
HD, IT15, LOMARS
Genomic regions
Chromosome 4
Summary
Huntingtin is a disease gene linked to Huntington's disease, a neurodegenerative disorder characterized by loss of striatal neurons. This is thought to be caused by an expanded, unstable trinucleotide repeat in the huntingtin gene, which translates as a polyglutamine repeat in the protein product. A fairly broad range of trinucleotide repeats (9-35) has been identified in normal controls, and repeat numbers in excess of 40 have been described as pathological. The huntingtin locus is large, spanning 180 kb and consisting of 67 exons. The huntingtin gene is widely expressed and is required for normal development. It is expressed as 2 alternatively polyadenylated forms displaying different relative abundance in various fetal and adult tissues. The larger transcript is approximately 13.7 kb and is expressed predominantly in adult and fetal brain whereas the smaller transcript of approximately 10.3 kb is more widely expressed. The genetic defect leading to Huntington's disease may not necessarily eliminate transcription, but may confer a new property on the mRNA or alter the function of the protein. One candidate is the huntingtin-associated protein-1, highly expressed in brain, which has increased affinity for huntingtin protein with expanded polyglutamine repeats. This gene contains an upstream open reading frame in the 5' UTR that inhibits expression of the huntingtin gene product through translational repression.
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
HTT
had been KO in hela cell line.
EZ-editor™ Gene Dependency
EZ-editor™ Gene Expression Level
EZ-editor™ Gene Copy Number
EZ-editor™ Gene Dependency
Result
The HTT gene you inquire is evaluated as high risk
in 5%
cell line.
Cell line is not selected, unable to assess the accurate risk level, for reference only.
In all cell lines, there is
33.8% cells with low expression level,
66.2% cells with medium expression level
of HTT gene.
Cell line is not selected, unable to assess the accurate expression level, for reference only.
In all cell lines, there is
72.7% cells with low copy number,
24.6% cells with medium copy number,
2.7% cells with high copy number
of HTT 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 
