Recombinant Virus

Adeno-associated Virus

Adeno-associated viruses (AAVs) belong to the Parvoviridae family, comprising a group of small-sized non-enveloped viruses with an icosahedral capsid carrying a single-stranded DNA genome of approximately 4.7 kilobases (kb). AAV is one of the most popular gene delivery vehicles due to its multiple, wide-ranging tropism profiles, low immunogenicity, long-term stable gene expression, and strong diffusion capabilities.

At OBiO, we can supply you a holistic vector design, gene synthesis, plasmid and AAV producing services. From idea to AAV, we can help you to delivery gene to any kind of cell you want to target, both in vivo and in vitro.



* We offer a variety of packaging systems for AAV vectors, in addition to the traditional Triple Plasmid System, we can also provide the dual plasmid system and sf9-baculovirus for Production of AAV, and we offer large scale AAV preps are produced in cell factory or shake flask.

*Available purification method: ultracentrifugation, chromatography


| Types of AAV we offered


*Single-stranded AAV(ssAAV) and self-complementary AAV(scAAV).

*100+ different serotypes available:AAV1-9, AAV-PHP.eB, AAV-PHP.S, AAV2-retro, AAV-ie, AAV-LungM3, AAV-LungX, AAV-GC01, AAV-GC02, AAV-GC03, AAV-GC13, AAV-DJ, AAV2.7m8, AAV6.2ff, AAV-PAN, AAVRec2, AAVrh10, custom AAV serotype

|Quality, scale and turnaround time


 

Research Grade

GMP-similar

Suitable for

Rodents, other small mammals

Dog, Pig, Sheep, NHP, other larger animals

Quality standard

Titer ≥ 1E+13vg/mL (with ddPCR/qPCR)

AAV Capsid determination:Match capsid protein size

Sterility test:Negative

Endotoxin Assay(optional):<10EU/mL

Mycoplasma Detection(optional):Negative

Titer ≥1E+13vg/mL(with ddPCR)

AAV Capsid determination:Match capsid protein size

Residual Nuclease:<0.1ng/ml

HCP:<20ng/ml

Purity Assay:>95%

Endotoxin Assay:<10EU/mL

Empty Capsid Rate:<10%

Sterility test:Negative

Mycoplasma Detection:Negative

Scale

multiple scales from1E+12vg to 5E+13vg

multiple scales from1E+13vg to 1E+15vg


* Guaranteed titers are suit for regular packaging, in which, (1) the payload gene size cannot exceed the AAV packaging capacity (4.7 kb from ITR to ITR), (2) you can choose AAV1, AAV5, AAV8, AAV9, AAV-retro, AAV-PHP.eB, AAV-PHP.S, AAV-ie, AAV-LungM3, AAV-LungX, AAV-DJ, AAV2.7m8, AAV-PAN, AAVRec2 or AAVrh10 to package your GOI.

* For big size gene packaging or low-yield AAV serotypes, or novel AAV, we can supply you the virus with typical titer > 5E+12 vg/mL, any needs could be inquired.

* For GMP-similar grade AAV producing, we can supply you AAV manufacturing with multi-serotypes, any needs could be inquired.

* Turnaround time depends on the complexity of gene synthesis, vector construction and kind of AAV serotype. Listed time is an estimated time. We will report you a confirmed time based on the technical proposal.


| Tissue-tropism of adeno-associated viral vectors


Serotype

Tissue tropism

AAV1

CNS, skeletal muscle, smooth muscle, retina, Endothelial Cell

AAV2

CNS, retina, liver, inner ear, kidney, smooth muscle

AAV3

muscle, liver, lung

AAV4

CNS, retina, muscle

AAV5

CNS, smooth muscle, retina, lung

AAV6

lung, CNS, smooth muscle, heart

AAV7

muscle, liver

AAV8

CNS, liver, kidney, adipose, retina, muscle, pancreas

AAV9

CNS, heart, lung, retina, skin

AAVDJ

retina, liver, kidney, in-vitro

AAV2-Retro

CNS:retrograde tracers

AAV9-PHP.B

CNS:crossed the blood-brain barrier

AAV9-PHP.eB

CNS:crossed the blood-brain barrier

AAV9-PHP.S

PNS:intravenous administration

AAV2-7m8

retina

AAV-LungX

pulmonary vascular endothelial cells

AAV-LungM3

lung

AAV-PAN

pancreas(intraperitoneal injection)

Anc80L65

inner ear, retina, skeletal muscle,liver

AAV6-ShH10Y

muller cells

AAV2-BR1

brain microvasculature endothelial cell

AAVRec2

adipose

AAV6-TM6

microglia

AAV6.2FF

lung, muscle

AAV2-rh10

CNS, liver, heart, lung, in-vitro

AAV-DSS

bone

AAV9-MyoAAV 1A

skeletal muscle, Cardiac muscle

AAV-Vec

vascular endothelial cells

AAV-ie

inner ear

AAV-ie-K558R

inner ear

AAV-DS1

inner ear

AAV-BI30

CNS: Endothelial Cells

AAV-MaCPNS1

PNS in rodent, PNS and CNS in macaque and marmoset (systemic administration)

AAV-MaCPNS2

AAV-MG1.2

microglia with high efficiency

AAV-GC01

retina:the retinal pigment epithelium (intravitreal injection)

AAV-GC02

retina:the retinal pigment epithelium (intravitreal injection)

AAV-GC03

retina (intravitreal injection)

AAV-GC13

retina: Cone and Rod (intravitreal injection)

* Please note, virus genome backbone is from AAV2, i.e. we use ITR2 for packaging.

Different serotypes are distinguished by the different capsid proteins.

 

| Promoter choices


For cell-specific gene expression, listed promoters can be designed into your vector.


Organ

Name

Cell Type Specificity

Brain/CNS

SYN1

Mature neurons

CaMKIIa

Pyramidal neurons

TH

Dopaminergic neurons

GFAP/GfaABC1D

Astrocytes

CX3CR1

Microglia

Iba1

Microglia

F4/80

Microglia

GAD67

GABAergic neurons

VGAT1

GABAergic neurons

mDLX

GABAergic neurons

fPV

Inhibitory neuron subtype (PV)

fSST

Inhibitory neuron subtype (SST)

fNPY

Inhibitory neuron subtype (NPY)

DAT

Dopaminergic neurons

L7pcp2

Purkinje cells

MBP

oligodendrocytes

NG2/NGL2

NG2-glia/oligodendrocyte precursor cells

PRSx8

adrenergic neurons/NE neurons

ChAT

cholinergic neurons

HCRT

Hcrt neurons

FEV

serotonergic neurons

Nestin

Neural stem cells (NSCs)

Retina

ProA1

Cone photoreceptor

hRHO

Rod photoreceptor

BEST1(VMD2)

RPE

Inner cells

IHC

Inner hair cells

OHC

Outer hair cells

HC

Hair cells

OPC

Outer pillar cells

IBC

Inner border cells

SC1

Supporting cells

SGN I

Type I neuron

SGN II

Type II neuron

Mammalian Inducible Promoters

TRE

tetracycline-inducible promoters

c-FOS

c-fos+ neurons/engram cells

Heart

cTnTo/cTnT

Cardiomyocytes

TCF21

Cardiac fibroblasts

Postn

Cardiac myofibroblast

Liver

Alb

Mature hepatocytes

TBG/Apoe

Hepatocytes

GFAP/α-SMA

Hepatic stellate cells

F4/80

Kupffer cells

Lung

SP-C

AT II cells (alveolar type II epithelial cells)

SP-B

AT II cells and Clara cells (bronchiolar epithelial cells)

CC10

Bronchial epithelial cells

Adipose

Adipoq

Adipocytes

Muscle

MHCK7

Differentiated postmitotic striated muscle cells

SM22a

Vascular smooth muscle cells

dMCK/tMCK

skeletal muscles

Myog

Myoblasts

Intestine

Villin

Intestinal epithelial cells

MUC2

Intestinal goblet cells

Pancreas

HIP/Mlp2/Ins2/Pdx1

Islet β-cells

GCG

Islet α-cells

ECs

hFLT1

Vascular endothelial cells

Tie1

Endothelial cells

Mammalian Ubiquitous Promoters

CMV

Strong promoter; may have variable strength in some cell types.

EF1A

Strong promoter.

EF1

Medium-strength promoter

EFS

Medium-strength promoter

CAG

Strong promoter

CBh

Strong promoter

SFFV

Strong promoter; drives high levels of gene expression, particularly in cell types of the myeloid lineage.

mPGK

Medium-strength promoter

hPGK

Medium-strength promoter

UBC

Weak promoter

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