Table 1:
Several natural and engineered AAV vectors tested in non-human primates for applications in the CNS
Variant | Parental serotype | Design | Tropism | Administration | Study |
---|---|---|---|---|---|
7m8 | AAV2 | 7-mer peptide insertion at AA588 (LGETTRP) | Pan-retinal | subretinal and intravitreal | 37 |
AAV1 and AAV2 | - | - | broad transduction of medium spiny neurons and cortico-striatal neurons | direct injection in caudate nucleus and putanem | 64 |
AAV9 | - | - | broad transduction, including retrograde and anterograde transport under local administration; <3% transduction of neurons and astrocytes by intravenous injection; ~2% transduction of brain and spinal cord after intrathecal injection | direct injection into parenchyma; intravenous; intrathecal | 65,66 |
PHP.B | AAV9 | 7-mer peptide insertion at AA588 (TLAVPFK) | <3% transduction of neurons and astrocytes | intravenous | 66 |
AAV5 | - | - | neurons and astrocytes | direct injection into parenchyma | 67 |