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 GBA Knockout
model in cell line by CRISPR-U™-mediated genome engineering.
Target gene info
Official symbol
GBA
Gene id
2629
Organism
Homo sapiens
Gene type
protein-coding
Official full symbol
glucosylceramidase beta
Also known as
GBA1, GCB, GLUC
Genomic regions
Chromosome 1
Summary
This gene encodes a lysosomal membrane protein that cleaves the beta-glucosidic linkage of glycosylceramide, an intermediate in glycolipid metabolism. Mutations in this gene cause Gaucher disease, a lysosomal storage disease characterized by an accumulation of glucocerebrosides. A related pseudogene is approximately 12 kb downstream of this gene on chromosome 1. Alternative splicing results in multiple transcript variants.
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
GBA
had been KO in 293t cell line.
EZ-editor™ Gene Expression Level
EZ-editor™ Gene Copy Number
EZ-editor™ Gene Expression Level
Result
In all cell lines, there is
2.3% cells with low expression level,
97.7% cells with medium expression level
of GBA gene.
Cell line is not selected, unable to assess the accurate expression level, for reference only.
In all cell lines, there is
45.5% cells with low copy number,
45.7% cells with medium copy number,
8.8% cells with high copy number
of GBA 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!