Gene therapy targeting synaptopathy linked with Alzheimer's and Parkinson's disease - PubMed
6 hours ago
- #Synaptopathy
- #Gene Therapy
- #Neurodegenerative Diseases
- Synaptic plasticity is crucial for memory formation and is a key research topic in neuroscience.
- Synaptopathy (altered synaptic plasticity) is linked to Alzheimer's (AD) and Parkinson's (PD) diseases.
- Amyloid-β (Aβ) aggregates and hyperphosphorylated tau contribute to synaptopathy in AD.
- Defective neurotransmitter release disrupts synaptic gene activity, protein function, and Ca2+ homeostasis.
- α-synuclein (ASN)-dependent synaptopathy is central to dopaminergic nerve injury in PD.
- Familial-PD is caused by mutations in genes like SNCA, LRRK2, Parkin, PINK1, and DJ-1.
- Familial AD is associated with variants in APP, PSEN1, and PSEN2 genes.
- CRISPR and AAV-based gene therapies show promise for tailored treatments in AD and PD.
- This review examines gene therapies targeting synaptopathy, amyloidopathy, and tauopathy in clinical and pre-clinical trials.