UC San Diego Looking For Volunteers In Gene Therapy Trials On Humans


The University of San Diego School of Medicine has launched a first-in-human Phase I clinical trial to test the benefits and safety of gene therapy. They are now looking for participants.

Researchers are using the trial to see if gene therapy can be used to deliver a key protein (brain-derived neurotrophic factor or BDNF) into the brains of those with Alzheimer's disease or Mild Cognitive Impairment, which often leads to dementia.

"We found that delivering BDNF to the part of the brain that is affected earliest in Alzheimer's disease - the entorhinal cortex and hippocampus - was able to reverse the loss of connections and to protect from ongoing cell degeneration," said Mark Tuszynski, professor and director of the Translational Neuroscience Institute at UCSD School of Medicine. "These benefits were observed in aged rats, aged monkeys and amyloid mice."

In the clinical trials, "UC San Diego researchers will inject harmless virus carrying a restorative gene into participants’ brains, where earlier animal studies suggest it may slow, prevent or reverse progression of the neurological disorder."

The trial will take place over three years and recruit 12 participants with diagnoses of Alzheimer's disease or Mild Cognitive Impairment to receive the treatment, and another 12 participants will serve as 'comparative controls during the trial.

You can find more information here.

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