human GAA Over-expression AAV

Cat. No: AAV-209485

Availability: 3-5 weeks

Name: AAV-h-GAA

This is an AAV expressing Human GAA.

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Viral Backbone: Recombinant AAV
AAV-ITR: AAV2
AAV Serotype: Available in AAV1, AAV2, AAV3, AAV5, AAV6, AAV8, AAV9, AAV-DJ, AAV-DJ8, AAV-DJ9 and other wildtype/synthetic AAV capsids
Promoter: CMV (default), or choose from 30 different ubiquitous or cell specific promoter
Reporter: none, optional GFP, CFP, YFP, RFP or mCherry
Storage Buffer: PBS/5% Glycerol

Species: Human
Gene Symbol: GAA
Gene Name: glucosidase alpha, acid
Gene ID: 2548
Gene Synonyms: LYAG
ORF Size: 2859 bp
RefSeq#: BC040431
Gene Description: 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.
UniGene ID: Hs.1437
HGNC ID: HGNC:4065