shRNA Adeno-associated Virus Serotype 2, p7SK-(C21orf2-shRNA-Seq1)(CAT#: AAV-SI1354WQ)

This product is a C21orf2-shRNA encoding AAV, which is based on AAV-2 serotype. The C21orf2 gene is down-regulated in Down syndrome (DS) brain, which may represent mitochondrial dysfunction in DS patients. The expression of C21orf2-shRNA leads to target gene silencing and this product can be used in gene therapy research and development.

Specifications
Family Parvoviridae
Species Adeno-associated virus (AAV)
Serotype AAV-2
Backbone AAV-2
Tropism CNS, muscle, liver, brain, eye
Insert C21orf2-shRNA-Seq1
Related Target/Protein C21orf2
Region 3UTR
TargetSeq GTTGTGACAGTCTTCCTGAAA
NCBI RefSeq NM_004928
Alternative Names RDMS; SMDAX; LRRC76; YF5/A2; CFAP410
Titer >1*10^10 GC/mL
Related Diseases Amyotrophic lateral sclerosis, Down syndrome (DS) brain
Target Gene
Gene ID 755
Uniprot ID O43822

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