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. Author manuscript; available in PMC: 2024 Mar 1.
Published in final edited form as: Wiley Interdiscip Rev Nanomed Nanobiotechnol. 2022 Oct 3;15(2):e1853. doi: 10.1002/wnan.1853

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