PPARγ-coactivator-1α gene transfer reduces neuronal loss and amyloid-β generation by reducing β-secretase in an Alzheimer’s disease model
File(s)PNAS.pdf (3.04 MB)
Accepted version
Author(s)
Type
Journal Article
Abstract
Current therapies for Alzheimer’s disease (AD) are symptomatic and do not target the underlying Aβ pathology and other important hallmarks including neuronal loss. PPARγ-coactivator-1α (PGC-1α) is a cofactor for transcription factors including the peroxisome proliferator-activated receptor-γ (PPARγ), and it is involved in the regulation of metabolic genes, oxidative phosphorylation, and mitochondrial biogenesis. We previously reported that PGC-1α also regulates the transcription of β-APP cleaving enzyme (BACE1), the main enzyme involved in Aβ generation, and its expression is decreased in AD patients. We aimed to explore the potential therapeutic effect of PGC-1α by generating a lentiviral vector to express human PGC-1α and target it by stereotaxic delivery to hippocampus and cortex of APP23 transgenic mice at the preclinical stage of the disease. Four months after injection, APP23 mice treated with hPGC-1α showed improved spatial and recognition memory concomitant with a significant reduction in Aβ deposition, associated with a decrease in BACE1 expression. hPGC-1α overexpression attenuated the levels of proinflammatory cytokines and microglial activation. This effect was accompanied by a marked preservation of pyramidal neurons in the CA3 area and increased expression of neurotrophic factors. The neuroprotective effects were secondary to a reduction in Aβ pathology and neuroinflammation, because wild-type mice receiving the same treatment were unaffected. These results suggest that the selective induction of PGC-1α gene in specific areas of the brain is effective in targeting AD-related neurodegeneration and holds potential as therapeutic intervention for this disease.
Date Issued
2016-10-25
Date Acceptance
2016-08-23
Citation
Proceedings of the National Academy of Sciences of USA, 2016, 113 (43), pp.12292-12297
ISSN
0027-8424
Publisher
National Academy of Sciences
Start Page
12292
End Page
12297
Journal / Book Title
Proceedings of the National Academy of Sciences of USA
Volume
113
Issue
43
Copyright Statement
© 2018 National Academy of Sciences.
Sponsor
Commission of the European Communities
Alzheimer's Research UK (ARUK)
Alzheimer's Research UK (ARUK)
Grant Number
Agreement no 233147
ART/PG2009/5
ARUK-ESG2013-8
Subjects
Science & Technology
Multidisciplinary Sciences
Science & Technology - Other Topics
A beta
BACE1
growth factor
inflammation
neurodegeneration
TRANSGENIC MOUSE MODEL
NERVE GROWTH-FACTOR
PPAR-GAMMA
TRANSCRIPTIONAL COACTIVATOR
ALS MODEL
MICE
PGC-1-ALPHA
EXPRESSION
NEURODEGENERATION
MEMORY
Aβ
Alzheimer Disease
Amyloid Precursor Protein Secretases
Amyloid beta-Peptides
Animals
Aspartic Acid Endopeptidases
Gene Expression Regulation
Genetic Vectors
Humans
Lentivirus
Memory
Mice, Transgenic
Peroxisome Proliferator-Activated Receptor Gamma Coactivator 1-alpha
Protein Aggregation, Pathological
Pyramidal Cells
MD Multidisciplinary
Publication Status
Published
Date Publish Online
2016-10-10