In scientific applications using adeno-associated virus (AAV) vectors, the choice of AAV serotype and promoter plays a crucial role in determining the efficiency, specificity, and duration of transgene expression. Different AAV serotypes exhibit varying tropisms for different cell types and tissues, while promoters regulate the expression of the transgene in a cell- or tissue-specific manner.
It has been found in study that AAV has multiple serotypes. The main difference between various serotypes of adeno-associated virus (AAV) vectors lies in the capsid proteins that make up the viral shell. These capsid proteins determine the tropism, or the ability of the virus to infect and transduce specific cell types or tissues. Different AAV serotypes have evolved to target different cell surface receptors and co-receptors, leading to variations in transduction efficiency and specificity in various cell types and tissues.
Researchers have identified and characterized numerous AAV serotypes with distinct capsid proteins, each with unique properties and tropisms. By utilizing different AAV serotypes, researchers can target specific cell types or tissues for gene delivery in gene therapy applications. For example, some AAV serotypes may have a preference for transducing neurons in the central nervous system, while others may be more efficient at targeting liver cells or muscle cells.
Tissue-specific promoters, also known as organ-specific promoters, play a crucial role in gene therapy and molecular biology research. These promoters regulate the expression of a gene in a specific tissue or organ, providing a means to target gene expression to particular tissues or organs while minimizing off-target effects. By using tissue-specific promoters, researchers can confine gene expression to the desired location, thereby enhancing the specificity and efficacy of gene regulation.
Tissue-specific promoters offer several advantages over constitutive promoters, such as the cytomegalovirus (CMV) promoter, which drive gene expression in a wide range of cell types. Constitutive promoters can lead to non-specific, continuous, and high levels of gene expression in various tissues, potentially causing unwanted side effects or toxicity. In contrast, tissue-specific promoters allow for precise control over gene expression, limiting it to specific cell types or organs where the regulation effect is needed.
Therefore, selecting appropriate serotypes with specific promoters for different tissues and organs enables targeted and efficient infection.
Injection site | serotype | Injection method |
Heart | AAV9 | In situ multi-point injection (topical) |
Tail vein injection (systemic) | ||
Liver | AAV8/AAV9 | Tail vein injection |
Whole brain (across the blood-brain barrier) | AAV-BBB | Caudal IV |
Brain (local) | AAV9/AAV2/AAV5 | Stereotaxic injection |
Retrograde tracer | AAV-Retro | Local injection |
Spinal cord | AAV5/AAV1/AAV9 | Intrathecal injection |
Fat | AAV9 | Intra-abdominal fat - intraperitoneal injection |
Subcutaneous fat. - It was an injection | ||
Skeletal muscle | AAV9 | In situ multiple injection |
Eyes | AAV2 | Vitreous cavity injection |
AAV-DJ | Subretinal cavity injection | |
Lungs | AAV6 | Non-invasive trachea injections/nasal drops |
Kidneys | AAV2/AAV9 | Renal pelvis in situ/caudal vein |
Enteric | AAV1/AAV5 | Enema/abdominal injection |
Blood vessels (endothelium predominant) | AAV1/AAV5 | Local infection of the abdominal aorta |
Pancreas | AAV8 | Intraperitoneal/tail vein injection |
Tissues | Promoter | Characteristics |
Neurons | hSyn | Neuron-specific promoter |
mecp2 | Shorter neuron-specific promoter | |
C-fos | Excitatory neuron promoter | |
CaMKIIα | 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 |
nphs2 | 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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