TABLE 3.
Clinical trials on nonviral nanoparticles for gene delivery to the brain
| Name | Indication | Therapeutic molecule | Type of nanocarrier | Clinical trial |
|---|---|---|---|---|
| Nusinersen | Spinal muscular atrophy | Antisense oligonucleotide targeting ISS-N1 | Antisense oligonucleotide | NCT04591678 |
| NU-0129 | Glioblastoma | siRNAs targeting Bcl2L12, a glioblastoma oncogene | Gold nanoparticles | NCT03020017 |
| BIIB094 | Parkinson’s disease | Antisense oligonucleotide targeting LRRK2 | Antisense oligonucleotide | NCT03976349 |
| ALN-APP | Alzheimer’s disease; cerebral amyloid angiopathy | RNAi targeting amyloid precursor protein (APP) | RNA interference | NCT05231785 |