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. 2025 Jan 17;22(3):e00525. doi: 10.1016/j.neurot.2025.e00525

Table 1.

Mitochondria AAV based therapeutics in Alzheimer's disease.

AAV-Protein Protein function Model Effects Reference
AAV9-NDI1 Yeast mitochondrial NADH dehydrogenase; electron transport chain; apoptosis SH-SY5Y Aβ1-42 ​cell model,
1-42 hippocampal injected mice.
Restored oxidative phosphorylation; reduces ROS production Li et al., 2024
AAV9-DJ-1 Regulation of apoptosis;
PINK1/parkin-mediated mitophagy;
Antioxidant response through NRF2 activation
APP/PS1 Enhanced cognitive function; increased levels of antioxidant genes (NRF2, SOD, GPx) Peng et al., 2023
AAV-Trx-1 Maintains redox homeostasis;
Antioxidant ROS scavenger
APP/PS1 Improved learning and memory;
Reduced hippocampal Aβ deposition; increased expression of APRK, Sirt1, PGC-1α
Jia et al., 2023
AAV-PGC-1α Regulator of oxidative phosphorylation;
Regulator of mitochondrial biogenesis
APP/PS1 Improved learning and memory;
Increase mitochondrial fusion protein (OPA1, MFN2); reduced mitochondrial fission protein (DRP1, FIS1)
Wang et al., 2022
AAV2-CEND1 Neuronal lineage-specific modulator;
Regulates mitochondrial dynamics via DRP1
5xFAD Improved learning and memory;
Increase mitochondrial fusion protein (OPA1, MFN2); reduced mitochondrial fission protein (DRP1)
Xie et al., 2022
AAV5-CHCHD6 MICO complex component;
Key component for mitochondrial crista formation
APPNL−F-G KI AD Reduced APP accumulation in the MAM; lowered C99 fragment; improved spatial working memory; restored synaptic function Shang et al., 2022
AAV9-MICU3 Regulates mitochondrial calcium uptake Tg-SwDI AD Improved behavioral performance; enhance cerebral blood flow; reduce Aβ deposition Zhou et al., 2023
AAV2-PINK1 Mitochondrial protein kinase;
Mitophagy
APPSwInd (J20) AD Alleviated mitochondrial dysfunction and oxidative stress; increased ATP;
Increased mitophagy; reduced Aβ accumulation
Du et al., 2017
AAV-MCL1 OMM LIR motif receptor;
Mitophagy;
Regulates apoptosis
APP/PS1 Improved learning and memory; reduced hippocampal Aβ deposition; Cen et al., 2020
DISC1 OMM LIR motif receptor;
Mitophagy;
Mitochondrial dynamics;
APP/PS1 Reduced expression of BACE1; reduced hippocampal Aβ deposition; improved learning and memory Deng et al., 2016