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Editorial
July 2018

Gene Therapy in Alzheimer Disease—It May Be Feasible, but Will It Be Beneficial?

Author Affiliations
  • 1Department of Neurology, Columbia University Vagelos College of Physicians and Surgeons, New York, New York
  • 2The Taub Institute for Research on Alzheimer's Disease and the Aging Brain, Columbia University Herbert and Florence Irving Medical Center, New York, New York
  • 3GH Sergievsky Center, Columbia University Herbert and Florence Irving Medical Center, New York, New York
JAMA Neurol. 2018;75(7):791-793. doi:10.1001/jamaneurol.2017.4029

In this issue of JAMA Neurology, Rafii et al1 report the results of a randomized clinical trial of intracerebral gene delivery in patients with Alzheimer disease (AD). Neurodegenerative diseases, of which AD is the most common, remain a challenging frontier for medical treatment. There are no preventive measures, and for the millions of individuals who already have AD, there are no disease-modifying agents to slow, stop, or reverse progression. The primary efforts of the field include a diverse assortment of ongoing drug trials attempting to alleviate or reduce various AD pathophysiologic processes: amyloidogenesis, neurofibrillary change, synaptic losses, and neuronal loss.2 While these efforts may be getting closer to success, they have not yet reached fruition.

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