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It has been found in study that AAV has multiple serotypes.The main difference between various different serotypes of AAV vectors is the difference in capsid protein, so transfection efficiency of different serotypes in different tissues and cells are differential.
Tissue-specific promoters are also called organ-specific promoters. Under the action of such a promoter, the expression of a gene is commonly confined to some particular tissue or organ, and at the same time it exhibits developmental regulation and other properties. In this way, the waste caused by non-specific, continuous and efficient expression of foreign genes activated by constitutive promoters can be overcome and the effect of gene regulation can be increased.
Therefore, selecting appropriate serotypes with specific promoters for different tissues and organs enables targeted and efficient infection.
| Tissues | Serotypes |
|---|---|
| Heart | AAV9 |
| Liver | AAV8/AAV9 |
| Brain (traverse the blood brain barrier) | AAV-BBB |
| Brain(partial delivery) | AAV9/AAV2/AAV5 |
| Retrograde delivery | AAV-Retro |
| Spinal Cord | AAV5/AAV1/AAV9 |
| Adipose Tissue | AAV9 |
| Skeletal Muscle | AAV9 |
| Eyes | AAV2/AAV-DJ |
| Lung | AAV6 |
| Kidney | AAV2/AAV9 |
| Intestine | AAV1/AAV5 |
| Blood Vessels (mainly endothelium) | AAV1/AAV5 |
| Pancrea | AAV8 |
| Tissues | Promoter | Characteristics |
|---|---|---|
| Neurons | hSyn | Neuron-specific promoter |
| mecp2 | Shorter neuron-specific promoter | |
| c-fos | Excitatory neuron promoter | |
| CaMKllα | Forebrain excitatory neurons | |
| Nms | Neural precursor cell-specific promoter | |
| TUBA1A (Tα1) | Early neuron-specific promoter | |
| hVGAT | GABAergic neuron/interneuron-specific promoter | |
| TH | Dopaminergic neuron-specific promoter | |
| gfaABC1D (minimal GFAP) | Astrocyte-specific promoter | |
| CD68 | Activated microglia-specific promoter | |
| ibal | Activated and resting microglia-specific promoter | |
| TMEM119 | Microglia-specific promoter | |
| CNP (NPPC) | Oligodendrocyte or Schwann cell-specific promoter | |
| MBP | Oligodendrocyte-specific promoter | |
| PLP | Oligodendrocyte-specific promoter | |
| Pdgfra | Oligodendrocyte precursor cell-specific promoter | |
| NG2 | Oligodendrocyte precursor cell-specific promoter | |
| Pirt | Sensory neuron-specific promoter | |
| ALDH1L1 | L1 Hypothalamic astrocyte-specific promoter | |
| Heart | cTNT | Cardiomyocyte-specific promoter |
| POSTN | Cardiac fibroblast-specific promoter | |
| TCF21 | Cardiac fibroblast-specific promoter |
|
| Muscle | MHCK7 | Muscle-specific promoter |
| SM22a | Smooth muscle-specific promoter | |
| MHCK7 | Muscle-specific promoter | |
| ACTA1 (synthesized Spc5-12) | Muscle cell-specific promoter | |
| MYOG |
Cardiac fibroblast-specific promoter | |
| Lung | SP-C (SFTPC) | Lung epithelial cell-specific promoter |
| Liver | TBG | Liver-specific promoter |
| Kidney | nphs1 | Kidney podocyte-specific promoter |
| nphs | Kidney podocyte-specific promoter | |
| Ksp-cadherin | Renal tubular epithelial cell-specific promoter | |
| Pancrea | padx1（pdx1） | Pancreas-specific promoter |
| Insulin2 | Pancreas-specific promoter | |
| Adipose Tissue | FABP4 (aP2) | Adipocyte-specific promoter |
| adiponectin | Adipocyte-specific promoter | |
| UCP1 | Brown adipocyte-specific promoter | |
| Myf5 | Adipocyte precursor cell-specific promoter | |
| Endothelium | TIE | Endothelial cell-specific promoter |
| Retina | rpe65 | Retina-specific promoter |
| Arr3 | Retinal cone cell-specific promoter | |
| hrk | Retinal cone and rod cell-specific promoter | |
| Prostate | DD3 | Prostate cancer cell-specific promoter |
| Skin | Keratin14（K14） | Skin epidermal cell-specific promoter |
| K14 | Keratin promoter | |
| Macrophages | F4/80 | Macrophage-specific promoter |
| Bone | COL2A1 | Chondrocyte-specific promoter |
| Stem Cells | Nanog | Pluripotent stem cell promoter |
| Serotype Name | Specific Cells |
|---|---|
| AAV-shH10 | Retinal Müller cells |
| AAV-Vec | Endothelial cells |
| AAV-MG | Microglia |
| AAV-MG1.2 | Microglia |
| AAV-br1 | Brain vascular endothelial cells |
| AAV-BI30 | Brain vascular endothelial cells |
| AAV-eVEC | Peripheral vascular endothelial cells |
| AAV-anc80L | Retina/Inner ear |
| AAV-ie | Inner ear |
| AAV-BBB | traverse the blood brain barrier |
| AAV-GL | Retina |
| AAV-NN | Retina |
| AAV-capB10 | Brain |
| AAV-BLS | Bladder |
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