Alan Verkman, MD, PhD
Professor Emeritus
Medicine
School of Medicine

Research Interests :

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Aquaporin water channels – Biophysics of cell membrane water permeability. Generation of transgenic mouse models to discover novel roles of aquaporins in tumor angiogenesis, cell migration, brain swelling, neuroexcitation, fat and tumor metabolism, skin hydration, saliva secretion, kidney tubule fluid transport, and others.

Cystic fibrosis – Drug discovery and elucidation of lung pathogenesis mechanisms. Developed screening methods to identify CFTR modulators, including the initial conceptualization and discovery of CFTR inhibitors, and potentiators and correctors of mutant CFTRs that cause cystic fibrosis.

Drug discovery and modeling of epithelial transport – High-throughput screening to identify small molecule modulators of epithelial chloride transporters, including CFTR, CaCCs, urea, and SLC26 proteins (DRA, pendrin, PAT1). Testing in animal models of secretory diarrheas, constipation, kidney stones, hypertension, edema, polycystic kidney disease, and dry eye disease.

Neuroinflammation / neuromyelitis optica spectrum disorders – Discovery of AQP4 pathogenesis mechanisms in NMOSD, including bystander complement and antibody-dependent cytotoxicity. Novel NMOSD therapeutics, including aquaporumab anti-AQP4 antibodies. Development of passive-transfer animal models of NMOSD.

Fluid and solute transport in cellular and extracellular compartments – Experimental measurements by photobleaching and single molecule methods using genetically targeted fluorescent proteins. Mathematical modeling of molecular crowding, diffusion and advection in cellular and extracellular spaces with recent application to the glymphatic hypothesis.

Assay and instrument development – Optical assays for drug screening including cell anion and volume sensors. Initial application of genetically encoded proteins to measure pH and Cl in cells. Microscopy developments including microfiberoptic & TIRFM photobleaching, astigmatic optics, single-particle tracking, tissue super-resolution imaging, and in vivo transport measurements.

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Google Scholars (h-index >175, citations > 100,000): https://scholar.google.com/citations user=8HhBMvQAAAAJ&hl=en

Patent portfolio (link to published US patents): https://patents.justia.com/inventor/alan-s-verkman

Global Health: Development of CFTR inhibitors for treatment of secretory diarrheas, including cholera, in developing countries. See news: https://innovation.ucsf.edu/news/fda-grants-orphan-drug-designation-vandas-cholera-treatment https://innovation.ucsf.edu/news/vanda-and-ucsf-announce-license-agreement-cftr-activators-and-inhibitors https://www.ucsf.edu/news/2006/11/102223/ucsf-discovery-major-focus-46-million-grant-combat-diarrhea https://www.nature.com/articles/news030106-16

Awards

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  • Verkman, Hans H. Ussing Award, American Physiological Society, 2025
  • Verkman, Paul Quinton Cystic Fibrosis Legacy Research Award, CFRI, 2019
  • Verkman, Robert J. Beall CF Therapeutics Development Award, Cystic Fibrosis Foundation, 2015
  • Verkman, NIH/NIBIB R37 MERIT award, 2007-2017
  • Verkman, NIH/NIDDK R37 MERIT award, 1999-2009
  • Verkman, Elected, Association of American Physicians (AAP), 1996
  • Verkman, Young Investigator Award, American Society of Nephrology and American Heart Association, 1993
  • Verkman, Elected, American Society for Clinical Investigation (ASCI), 1989
  • Verkman, Established Investigator, American Heart Association, 1988
  • Verkman, M.D. magna cum laude, Harvard Medical School, 1981
  • Verkman, National Science Foundation fellowship in Physics, Harvard University, 1975

Education & Training

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  • Residency Medicine Brigham & Womens Hospital
  • M.D. Harvard Medical School / Harvard-M.I.T. Program in Health Sciences and Technology
  • M.A., Ph.D. Physics Harvard University
  • S.B., S.B. Physics, Biology M.I.T.
  • Fellowship Nephrology University of California, San Francisco

Grants and Projects

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Publications (701)

Top publication keywords:
Aquaporin 3Immunoglobulin GBrain EdemaAquaporin 1Neuromyelitis OpticaDiarrheaIon ChannelsChloride ChannelsWaterCystic FibrosisAquaporin 4AquaporinsCystic Fibrosis Transmembrane Conductance RegulatorChloridesCell Membrane Permeability

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