Scientific Advisory Board

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Don W. Cleveland

  • Chair, Department of Cellular and Molecular Medicine (UCSD)
  • Distinguished Professor Of Cellular and Molecular Medicine, Medicine, and Neurosciences (UCSD)
  • Member, Ludwig Institute for Cancer Research

 

Dr. Cleveland has made pioneering discoveries of the mechanisms of chromosome movement and cell-cycle control during normal cellular division, as well as of the principles of neuronal cell development and their relationship to the defects that contribute to inherited neurodegenerative disease. Cleveland's research looks at the molecular genetics of axonal growth and motor neuron disease and the cell biology of mammalian chromosome movement.

Most recently, his research has achieved a significant breakthrough in treating Huntington's disease, an inherited and degenerative brain disorder for which there is no cure. A one-time injection of a new DNA-based drug treatment - known as ASO (short for antisense oligonucleotide) - blocked the activity of the gene whose mutation causes the disease. A single treatment silenced the mutated gene responsible for the disease, slowing and partially reversing progression of the fatal neurodegenerative disorder in animal models. This drug, called IONIS-HTTRx, was developed by scientists at Ionis Pharmaceuticals in collaboration with partners CHDI Foundation, Roche Pharmaceuticals and academic collaborators at University of California, San Diego and is now in a Phase 1/2a clinical study.

iXCells is working with Dr. Cleveland to study the mechanism of an ALS-associated protein TDP-43 using iPSC-derived motor neurons as the disease model.

Awards and Honors

  • The Breakthrough Prize in life sciences or mathematics, 2018
  • Elected Member, National Academy of Sciences, 2006
  • Elected Fellow, American Academy of Arts and Sciences, 2006
  • Elected Fellow, American Association for the Advancement of Science, 2009
  • Elected Member, The National Academy of Medicine, 2012
  • Elected Fellow, American Academy of Microbiology, 2006
  • President, American Society for Cell Biology, 2013
  • Katharine Berkan Judd Award, Memorial Sloan Kettering, 2012
  • Wings Over Wall Street and MDA Outstanding Scientist, October 2007
  • Outstanding Scientist Award, Playing to Win for Life Foundation, September 2004
  • Sheila Essey Prize, American Academy of Neurology and the ALS Association, April, 1999
  • NIH Jacob Javits Neuroscience Investigator MERIT Award from the National Institute of Neurologic Disease and Stroke, 2005-2012
  • NIH Jacob Javits Neuroscience Investigator MERIT Award from the National Institute of Neurologic Disease and Stroke, 1989-1996
  • NIH MERIT Award from National Institute of General Medicine, 1994-2004
  • NIH Research Career Development Award, 1982-1987
  • Chaim Weizmann Postdoctoral Fellow, 1978-1980
  • NSF Predoctoral Fellowship, 1972-1975
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Pengzhe (Paul) Lu

  • Adjunct Professor, UCSD
  • Header of spinal cord injury research group, Center For Neural Repair, UCSD
  • Member of Ad Hoc Committee, Department of Neurosciences, UCSD

 

Dr. Paul Lu received his Ph. D. in molecular biology from the University of California – Davis, and performed his post-doctoral research in the laboratory of Dr. Mark Tuszynski at the University of California – San Diego (UCSD). 

 

Dr. Lu then joined the faculty of University of California, San Diego, and is currently an Adjunct Professor of Neuroscience and a Research Health Science Specialist at the Veterans Administration San Diego Healthcare System. Dr. Lu’s research focuses on neural stem cells to form new neural relay circuits after spinal cord injury. Dr. Lu’s work aims to “splice” the injured neural circuit by implanting neural stem cells into sites of spinal cord injury.

iXCells is working with Dr. Lu to explore the clinical applications of hiPSC-derived motor neurons in regenerative medicine to treat motor neuron diseases, such as Amyotrophic Lateral Sclerosis (ALS).