Hanheng Biotechnology (Shanghai) Co., Ltd.
Specific Adeno-associated Virus Packaging

Specific Adeno-associated Virus Packaging

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. 

Specific Adeno-associated Virus Packaging Introductions

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.


AAV Serotype and Injection Method Recommended by Hanbio

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


Tissue-specific Promoters

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


AAV Serotypes for Specific Tissues

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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