human RPE65 shRNA silencing AAV

Cat. No: shAAV-221550

Availability: 6-8 weeks

Name: AAV-h-RPE65-shRNA

This is an AAV expressing shRNA for silencing of Human RPE65.

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Knockdown: shRNA screening service available, please inquire about this service or other options.

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: U6
Reporter: eGFP (default), optional CFP, YFP, RFP, mCherry
Storage Buffer: PBS/5% Glycerol

Species: Human
Gene Symbol: RPE65
Gene Name: RPE65, retinoid isomerohydrolase
Gene ID: 6121
Gene Synonyms: BCO3; LCA2; mRPE65; rd12; RP20; sRPE65
ORF Size: 1602 bp
RefSeq#: NM_000329
Gene Description: RPE65 encodes a protein which is located in the retinal pigment epithelium and is involved in the production of 11-cis retinal and in visual pigment regeneration. There are two forms of this protein, a soluble form called sRPE65, and a palmitoylated, membrane-bound form known as mRPE65. mRPE65 serves as the palmitoyl donor for lecithin retinol acyl transferase (LRAT), the enzyme that catalyzes the vitamin A to all trans retinol step of the chromophore regeneration process. Both mRPE65 and sRPE65 also serve as regulatory proteins, with the ratio and concentrations of these molecules playing a role in the inhibition of 11-cis retinal synthesis. Mutations in this gene have been associated with Leber congenital amaurosis type 2 (LCA2) and retinitis pigmentosa.
UniGene ID: Hs.2133
HGNC ID: HGNC:10294