Aquaporins in Neurological Disease

Date/Time: Monday, October 5, 2020 - 9:15 AM – 11:15 AM
Track: Plenary Session
Room: San Francisco
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In 2003, Dr. Peter Agre was awarded the Nobel Prize in Chemistry for his work leading to the discovery of aquaporin channels, which are responsible for moving water through cell membranes and are present in many species of plants and animals. Aquaporin 4 (AQP4) is heavily expressed on astrocyte foot processes and is implicated in the maintenance of the blood-brain-barrier, as well as in regulation of extracellular volume, cerebral spinal fluid circulation, waste clearance, cell migration, calcium and potassium signaling and neuroinflammation. Recent data suggest that AQP4 may play a role in the glymphatic (glial lymphatic) system, which is responsible for the elimination of soluble proteins and metabolites from the central nervous system. While AQP4 represents one of the most common molecules in the brain, only recently has its role in a variety of neurological diseases been appreciated. A wide spectrum of disorders have been linked to AQ4 including: neuromyelitis optica, hydrocephalus, cerebral edema following ischemic stroke or traumatic brain injury, glioma related vasogenic edema, cerebral malaria and neurodegenerative diseases such as Alzheimer Disease, Parkinson’s Disease and ALS. Recently, several immunotherapies have been developed to combat AQP4 related neuromyelitis optica. Understanding the underpinnings of the role of aquaporins in blood brain barrier maintenance and the glymphatic system may lead to more sophisticated and targeted therapies for a variety of CNS diseases. 

Chair: Jennifer Frontera, MD, NYU Langone
Co-chair: Elizabeth Silberman, MD, Oregon Health & Science University


  • To understand the role of aquaporin channels in maintenance of the blood brain barrier and glymphatic system 

  • To identify diseases related to AQP4 

  • To identify AQP4 related targets for therapeutics 


Aquaporin-4 in Neuromyelitis Optica

Keynote Lecture by Dr. Peter Agre


PRES and Aquaporins: Clinical Implications

Regulation of the Blood-Brain Barrier in Health and Disease

The Glymphatic System in Neurologic Disease