Table 3.
Representative promising therapeutic targets for gene therapy of neurodegenerative disorders.
| Target selection | Disorder | Gene therapy | Delivery route | Ref. |
|---|---|---|---|---|
| ER stress and UPR | Traumatic optic nerve injury | AAV2-XBP-1 | Intravitreal injection | 81 |
| Parkinson's disease | AAV2-XBP-1 | Unilateral brain injection | 82 | |
| Optic neuritis and encephalomyelitis | AAV2-CHOP shRNA | Intravitreal injection | 83 | |
| Parkinson's disease | AAV5-Bip | Intracerebral injection | 84 | |
| Amyotrophic lateral sclerosis | AAV6-SIL1 | Unilateral brain injection | 85 | |
| Huntington's disease | AAV2-XBP-1 | Intrastriatal injection | 86 | |
| mTOR signaling | Optic nerve injury | AAV2-AKT | Intravitreal injection | 87 |
| Optic nerve injury | AAV2-S6K1 | Intravitreal injection | 88 | |
| Optic nerve injury | AAV2-PTEN | Intravitreal injection | 89 | |
| Parkinson's disease | AAV1-AKT | Intrastriatal injection | 90 | |
| Alzheimer's disease and Parkinson's disease | AAV1-AKT | Intrastriatal injection | 91 | |
| Huntington's disease | AAV1-caRheb | Unilateral brain injection | 92 | |
| Mitochondrial function | Parkinson's disease | AAV2-HSP70 | Substantial nigra dense area injection | 93 |
| Alzheimer's disease | AAV2-PINK1 | Intrahippocampal injection | 94 | |
| Epigenetic regulation | Alzheimer's disease | AAV2-PSD95-6ZF-VP64 | Intrahippocampal injection | 95 |
| Autophagy | Alzheimer's disease | AAV2-PINK1 | Intrahippocampal injection | 94 |
| Parkinson's disease | AAV6-Lamp2a | Intrastriatal injection | 96 | |
| Parkinson's disease | AAV2-TFEB | Unilateral brain injection | 97 | |
| Amyotrophic lateral sclerosis | AAV9-snapin | Intravenous injection | 98 | |
| Microglial and astrocyte function | Alzheimer's disease | AAV2/8-sTREM2 | Intracerebral injection | 99 |
| Alzheimer's disease | Lentivirus-PGRN | Unilateral brain injection | 100 |