UC San Diego begins human trial to test gene therapy for Alzheimer's

Researchers from the School of Medicine at University of California, San Diego, have reportedly launched the first-in-human Phase I clinical trial assessing the safety and efficacy of gene therapy for the treatment of Alzheimer's disease or Mild Cognitive Impairment, a condition that often appears before full-developed dementia.

According to reliable sources, gene therapy involves delivering a key protein into the brains of patients with Alzheimer's disease (AD). This protein named brain-derived neurotrophic factor (BDNF) reportedly belongs to the family of growth parameters found inside the brain as well as central nervous system, which supports the survival of existing neurons and promotes both growth and differentiation of new synapses and neurons. BDNF is crucial in brain regions that are prone to degeneration in AD.

In a previously published research, Mark Tuszynski, MD, PhD, Director of Translational Neuroscience Institute and Professor of neuroscience at UC San Diego, stated that early studies observed the positive effects of gene therapy within aged monkeys, aged rats, as well as amyloid mice.

Tuszynski described that amyloid mice are genetically modified in order to inherit a mutation while gene encoding the amyloid precursor protein, and subsequently developing amyloid plaques, which are the aggregates of misfolded proteins present in the brain that are regarded as a hallmark trait of AD.

BDNF is usually produced through the entire life in the entorhinal cortex, a vital memory center inside the brain, one of the first regions where the effects of AD are typically observed in the form of short-term memory loss.

However, BDNF being a big size molecule cannot pass through the blood-brain barrier, making it complicated to work with. Considering this, researchers have shifted their focus towards gene therapy, which involves modifying a harmless adeno-associated virus (AAV2) for carrying the BDNF gene and injecting directly into targeted regions of the brain, where researchers estimate that it can stimulate production of therapeutic BDNF in nearby cells.

Tuszynski stated that gene therapy, which appeared first in 1980s, has been tested for the treatment of several diseases and conditions. It represents a different approach to an illness that call for new approached of thinking about it along with new attempts at its treatment.

The researchers are looking forward to making development based on the current successes of gene therapy in other diseases. For instance, the treatment of spinal muscular atrophy and Leber Hereditary Optic Neuropathy, Tuszynski added.

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