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. 2024 Nov 24;25(23):12613. doi: 10.3390/ijms252312613

Table 3.

Therapeutic innovations and clinical trials in neurodegenerative disease treatments.

Therapeutic Approach Molecular Target Disease Focus Therapeutic Agent(s) Trial Phase Key Outcomes Cited Studies
Nanotechnology-based drug delivery Blood–brain barrier penetration, tau/amyloid-beta targeting Alzheimer’s, Parkinson’s Liposomal encapsulation, PEG-modified nanoparticles Phase II (various) Improved brain bioavailability, reduced plaques/tangles in animal models Hajjo et al. (2022) [473], Saraiva et al. (2016) [474]
Stem cell therapy Dopaminergic neurons, motor neurons Parkinson’s, ALS iPSCs, MSCs Phase I/II Motor symptom improvement, neuron survival post-transplant Takahashi et al. (2007) [475], Glass et al. (2016) [476]
Gene therapy SOD1, HTT, APOE ε4 ALS, Huntington’s, AD Antisense oligonucleotides, CRISPR Phase I/II Reduced toxic protein levels, slower disease progression Kordasiewicz et al. (2012) [477], Bennett et al. (2019) [478]
Pharmacogenomics CYP2D6, COMT, APOE ε4 Alzheimer’s, Parkinson’s Genotype-guided dosing for existing drugs Phase II/III Reduced side effects, improved therapeutic outcomes Singh et al. (2017) [479], Müller et al. (2015) [480]
Microbiome modulation Gut–brain axis, inflammation Parkinson’s, Alzheimer’s Probiotics, prebiotics, antibiotics Phase I Reduced neuroinflammation markers, improved cognitive/motor symptoms Sampson et al. (2016) [481], Cattaneo et al. (2017) [482]