GAA Easy KO Kit

Name: GAA Easy KO Kit
SKU: CC-546
Availability: InStock

For research use only. Not intended for any clinical use.

Cat. No. : CC-546

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Product Information

Gene Information

Cat. No.	CC-546
Description	A complete kit for efficient gene knockout in mammalian cells, combining chemically synthesized sgRNAs with Cas9 RNPs to induce targeted DNA cleavage and generate frameshift mutations or deletions. All essential reagents for transfection and knockout validation are included for rapid, high-efficiency gene disruption.
Gene Abbr	GAA
Species	Human
EnsemblID	ENSG00000171298
NCBIGeneID	2548
Uni ProtID	P10253
Features	All-in-One workflow from gene editing to knockout validation for users with no prior experience. Pre-validated sgRNAs and primers for rapid setup. Streamlined experiment handling. CRISPR RNP method ensures precise and efficient gene knockout.
Applications	This kit enables in vitro gene knockout in human-derived cells using chemically synthesized sgRNAs and Cas9-gRNA RNP complexes. Transfected RNPs cleave early exons of the target gene, inducing deletions or frameshift mutations for efficient and rapid knockout.
Reactions	5–10 reactions per target gene
Kit Components	2–3 chemically synthesized sgRNAs (200pmol each) 3 PCR/Sequencing primers (500pmol each) LM cell lysate (500µL) Cas9 protein (12µg) LM RNP transfection reagent (50µL)
Storage	Store at -80°C for up to 1 year or at -20°C for up to 6 months. Avoid repeated freeze-thaw cycles.

Target Gene

GAA

Background

This gene encodes lysosomal alpha-glucosidase, which is essential for the degradation of glycogen to glucose in lysosomes. The encoded preproprotein is proteolytically processed to generate multiple intermediate forms and the mature form of the enzyme. Defects in this gene are the cause of glycogen storage disease II, also known as Pompe's disease, which is an autosomal recessive disorder with a broad clinical spectrum. Alternative splicing results in multiple transcript variants. [provided by RefSeq, Jan 2016]

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