New, experimental gene therapy is being tested on mouse models to potentially preserve learning and memory functions in Alzheimer's Disease patients.

Scientists at the University of California - San Diego School of Medicine have released findings of their study, "Synapsin-caveolin-1 gene therapy preserves neuronal and synaptic morphology and prevents neurodegeneration in a mouse model of AD," which is an immense step forward in realizing a practical treatment for humans suffering from the degenerative disease.

Alzheimer's Disease
(Photo: Mikael Häggström, M.D./Wikimedia Commons)
Histopathology of neurofibrillary tangles in the hippocampus in Alzheimer's disease.

People, mostly the elderly, are afflicted with Alzheimer's Disease due to accumulated masses of misfolded proteins that are called amyloid plaques and neurofibrillary tau tangles. These impair cell signaling and lead to neuronal death. Currently, treatments for Alzheimer's Disease target plaques and tangles, which would only address the symptoms of the illness.

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Researchers suggest that a reversal and remedy for Alzheimer's Disease would need a combination of interventional methods that both reduce aggregating toxins and boost neuronal and synaptic plasticity, a MedicalXpress article said.

Administering Gene Therapy SynCav1 to Alzheimer's Disease Mouse Models

Their gene therapy for Alzheimer's Disease is founded on introducing a therapeutic compound to a specifically targeted area of the brain that may bring back or safeguard normal neural functions and reverse neurodegenerative processes. For this particular case, researchers utilized a harmless adeno-associated viral vector to take the gene therapy Synapsin-Caveolin-1 cDNA (AAV-SynCav1) right into the hippocampus area of three-month-old transgenic mice with Alzheimer's Disease.

The mice were modified genetically to show learning and memory shortages at nine and 11 months. These learning and memory shortages were brought about by a reduced expression of Caveolin-1, the scaffolding protein that creates membranes that house cellular signaling tools. These include neurotrophin receptors that accommodate critical extracellular signals that rule cellular life and their capabilities. Damaged membranes would result in cell dysfunction and neurodegeneration.

The researchers' goal was to test if the SynCav1 gene therapy in the Alzheimer's Disease mouse models would keep neuronal and synaptic plasticity in targeted areas of the membrane and enhance brain functions, a Neuroscience News report said.

Neuronal, Synaptic Plasticity Preserved

Findings showed that such plasticity was indeed preserved after a single injection of AAV-SynCav1 to the hippocampus, a complex area deep inside the brain that plays a big part in providing memory and learning functions. In Alzheimer's Disease, the hippocampus is the most affected and impaired region of the brain.

At nine and 11 months, researchers said, hippocampal learning and memory were preserved. In addition, researchers also discovered that critical membrane structures and related neurotrophin receptors have likewise remained intact. Moreover, such neuroprotective SynCav1 gene therapy effects happened amidst the decrease in amyloid plaque depositions.

SynCav1 Gene Therapy, Promising Treatment for Alzheimer's Disease

The researchers said the SynCav1 therapy is truly a promising treatment to bring back brain plasticity and enhance brain functions in Alzheimer's Disease patients and possibly other types of neurodegenerative diseases.

They are currently experimenting with SynCav1 gene therapy in other Alzheimer's Disease models at symptomatic levels and mouse models of amyotrophic lateral sclerosis (Lou Gehrig's Disease). They hope to push their work for clinical trials at the soonest possible time.

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