human GAA shRNA silencing AAV

Name
AAV-h-GAA-shRNA

Cat No
shAAV-209485

Availability
4-5 weeks

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.

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Product Details

AAV Serotype

Available in AAV1, AAV2, AAV3, AAV5, AAV6, AAV8, AAV9, AAV-DJ, AAV-DJ8, AAV-DJ9 and other wildtype/synthetic AAV capsids

AAV ITR

AAV2

Promoter

U6 ()

Reporter

eGFP (default), optional CFP, YFP, RFP, mCherry

shRNA Knockdown (%)

Storage Buffer

PBS/5% Glycerol

Viral Backbone

Recombinant AAV

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

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.