CGT Wiki

Tail vein (IV)

1×10^11-1×10^12 vg/piece (about 5×10^12-5×10^13 vg/kg)

CNS targeting AAV9 3×10^13-1×10^14 vg/kg

1×10^12-6×10^13 vg/kg (system IV)

100-300 μL

2-6 weeks

Retroorbital vein (RO)

1×10^11-2×10^12 vg/only

Generally not recommended

—

100-150 μL

2-6 weeks

Portal vein (PV)

1×10^10-1×10^12 vg/only

1×10^11-1×10^12 vg/only

~10^12-10^13 vg/kg (historical plan, to be verified)

≤100 μL

2-6 weeks

Intraperitoneal cavity (IP)

1×10^11-1×10^12 vg/only

2×10^11 vg/only

—

100-200 μL

2-6 weeks

Subcutaneous (SC)

5×10^10-4×10^11 vg/only

To be verified (approximately 1×10^12 vg/kg level)

~10^12 vg level/part (to be verified)

~100 μL/dot

≥6 months

Intramuscular (IM)

1×10^9-1×10^11 vg/muscle; total multiple muscles 1×10^11-1×10^12

~1×10^11 vg/muscle

rhesus IM AAV8 3×10^12 GC/kg

25-50 μL/dot

2-6 weeks

Brain stereotaxy

1×10^9-1×10^10 vg/site

2×10^10-6×10^10 vg/site

1.5×10^11 vg/brain (putamen)

0.2-2 μL/site

3-4 weeks

Ventricles (neonatal/adult ICV)

Newborn 1-2×10^10 GC/hemisphere; adult 1×10^11 vg/side

Stereotaxy of adult lateral ventricles

4×10^13 vg (bilateral)

Newborn 1-2 μL/side; adult 5-10 μL

2-4 weeks

Intrathecal (IT/lumbar puncture)

5×10^10-1×10^11 vg/only

1-2×10^11 vg

4.8×10^12-9.2×10^13 vg/piece; same level as dog/pig

5-10 μL

3-6 weeks

Cerebellomedullary cistern (CM/ICM)

1×10^10-7×10^10 vg

1×10^12 vg

1.7-5×10^13 GC

1-10 μL

3-4 weeks

Lumbar puncture (LP)

Same as IT(L5-L6)

Same as IT

1×10^13 vg/kg; pig 1.4×10^13 GC/mL×400-800 μL

5-10 μL

3-6 weeks

Subretinal (SR)

1×10^8-3×10^9 vg/eye

1×10^9-1×10^10 vg/eye

7×10^11-7×10^12 vg/eye (AAV8); dog 1-1.4×10^11

1-2 μL

3-6 weeks

Intravitreal cavity (IVT)

1×10^9-1×10^10 vg/eye

1×10^9-5×10^9 vg/eye

7×10^11 vg/eye

1-2 μL

3-6 weeks

Intranasal/Intratracheal

4×10^11-1×10^12 vg/only

1×10^11-5×10^12 vg/only

NHP atomization ~3 mL; sheep 10 mL/time

25-50 μL

1-2 weeks

Renal vein/retrograde ureter

1×10^11-1×10^12 vg/kidney

1×10^11-5×10^11 vg/kidney

Data is limited

100 μL/kidney

2-4 weeks

Pancreatic duct

2×10^11-2×10^12 vg/only (AAV6)

To be approved

Limited data (ERCP path)

100 μL

4-6 weeks

Articular cavity (IA)

0.5×10^10-1×10^11 vg/joint

1×10^10-2.5×10^10 vg/joint

Clinical magnitude of equine OA

5 μL/joint

2-4 weeks

Intratumoral (ITu)

5×10^10-1×10^11 vg/tumor

1×10^10-5×10^10 vg/tumor

Spontaneous tumor in dogs (to be diagnosed)

50-100 μL/tumor

1-2 weeks

Sciatic nerve/peripheral nerve

2.1×10^8 vg(scAAV1)

2×10^11 GC/nerve

2.5×10^12-2.6×10^13 vg/nerve

2-20 μL

3-4 weeks

Cochlea/inner ear

1×10^10-5×10^10 vg/ear

About 2-5 μL (to be nucleated)

1×10^11-7×10^11 vg/ear

0.6-2 μL

2-4 weeks

Myocardial part

5×10^10-1×10^11 vg/heart

1×10^11-5×10^11 vg/heart

Canine 7-Fr endocardial catheter; pig multi-site thoracotomy

30 μL(3 points)

3-4 weeks

Fat pad

1×10^11-2×10^11 vg/pad

To be approved

—

30-50 μL

3-4 weeks

Intracutaneous/wound

~2×10^10 vg/dot

50-100 μL per point

Pig skin transformation model

10-20 μL/dot

2-4 weeks