human GAA shRNA silencing AAV
This is an AAV expressing shRNA for silencing of Human GAA.
shAAV-209485
AAV-h-GAA-shRNA
Ready-to-use AAV expressing shRNA for silencing of Human GAA (glucosidase alpha, acid). Available with optional GFP reporter or cell-specific promoter.
Gene Reference Data
Alternate Names
LYAG
Description (Vector)
GAA encodes acid alpha-glucosidase, which is essential for the degradation of glycogen to glucose in lysosomes. Different forms of acid alpha-glucosidase are obtained by proteolytic processing. 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. Three transcript variants encoding the same protein have been found for this gene.
Gene ID
2548
Gene Name (Vector)
glucosidase alpha, acid
Gene Symbol
GAA
HGNC ID
HGNC:4065
ORF Size (aa)
2859
ORF Size (bp)
2859 bp
RefSeq ID
NM_000152
RefSeq Synonyms
NM_001079804, NM_001079803, NM_000152, BC040431,
Species
human
UniGene ID
Hs.1437
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About Storage Conditions
All our viral products should be kept at -80°C. At this temperature, the virus will remain stable for 6-12 months (and in some cases, up to 2 years). Once thawed, the product can be stored at 4°C for 2-3 weeks without significant loss of biological activity.
We recommend aliquoting your vectors into low protein binding tubes upon receipt. This helps avoid repeated freeze-thaw cycles, as well as prevent loss of virus. To maintain accurate titer, aliquot in at least 20ul per tube.