Tissue selection adopts the combined model of "capsid entry × administration reachability × transcriptional regulation × dose × detection endpoint". The serotypes and doses in the table below are common starting points for preliminary experiments in mice; changes in disease model, age, and manufacturing processes may alter results.
Tissue serotype and promoter quick check
|
Organization |
Common natural/engineered candidates |
Commonly used transcriptional controls |
Common Paths |
|
Retina |
AAV2, AAV5, AAV8 and eye engineering capsids |
RPE65/VMD2, GRK1, SNCG |
Subretinal, vitreous cavity |
|
Liver |
AAV8, AAV5, AAV3B |
TBG, hAAT, LP1 |
Veins |
|
Heart |
AAV9, AAV6 |
cTnT/TNNT2, MYL2, MHCK7 |
Multiple points in veins and myocardium |
|
Skeletal muscle |
AAV9, AAVrh74, MyoAAV/AAVMYO |
MHCK7, desmin, CK8 |
Intravenous, intramuscular |
|
Bone/Cartilage |
AAV9, AAV-DJ or capsid screening |
COL1A1, BGLAP, COL2A1 |
Local, intravenous |
|
Lungs |
AAV5, AAV6, AAV9 and lung-targeting capsids |
SFTPC, SCGB1A1, FOXJ1 |
Intranasal, intratracheal, intravenous |
|
kidney |
AAV9, AAV2/8 (path dependency) |
NPHS1/2, CDH16, AQP2 |
Renal vein, retrograde ureter |
|
Pancreas |
AAV6, AAV8, AAV9 |
INS1/2, GCG, PDX1 |
Pancreatic duct, vein |
|
Fat |
AAV8, AAV9 and capsid screening |
ADIPOQ, UCP1, FABP4 |
Fat pad, vein |
|
Skin |
AAV2, AAV6, AAV8/9 |
KRT14, KRT5, IVL, COL1A1 |
Intradermal, local wound area |