Blau, Helen Margaret, Ph.D.
Donald E and Delia B. Baxter Professor
Director of the Baxter Laboratory for Stem Cell Biology
Department of Microbiology and Immunology & Institute for Stem
Cell Biology and Regenerative Medicine
Stanford University School of Medicine
EDUCATION
  • 1969 B.A. University of York, York, England
  • 1970 M.S. Harvard University, Cambridge, MA
  • 1975 Ph.D. Harvard University, Cambridge, MA
Research
Dr. Helen Blau received her B.A. from University of York in England and her M.A. and Ph.D. from Harvard University. She is currently the Donald E. and Delia B. Baxter Professor and Director of the Baxter Laboratory for Stem Cell Biology in the Microbiology and Immunology Department and the Stanford Institute for Stem Cell Biology and Regenerative Medicine in the Stanford University School of Medicine, Stanford, California.

Dr. Blau has been recognized in many ways, the most notable being: elected to the American Association for the Advancement of Science (1991), the Senior Career Recognition WICB Award, American Society of Cell Biology (1992), elected President, American Society for Developmental Biology (1994), Nobel Forum Lecture, Karolinska (1995), elected to Institute of Medicine, National Academy of Sciences (1995), elected to the American Academy of Arts and Sciences (1996), FASEB Excellence in Science Award (1999), elected President, International Society of Differentiation (2002-04); Honorary Doctorate, University of Nijmegen (2003); elected Harvard Board of Overseers (2004-2010), AACR-Irving Weinstein Foundation Distinguished Lecturership for outstanding innovations in science (2011).

Helen Blau is a pioneer in regenerative medicine. She is world-renowned for her seminal discovery that the differentiated state is reversible, rather than fixed and terminal. Her demonstration of cellular plasticity constituted a paradigm shift in our understanding of mammalian cell differentiation. Using muscle as a model, Blau’s work provided the first definitive evidence that diverse cell types could be reprogrammed using non-dividing fused cells (heterokaryons), overcoming problems of chromosome loss in previous hybrid studies and showing that John Gurdon’s findings of cellular plasticity are not limited to amphibians. Her work demonstrated that cell differentiation requires continuous regulation, and that a shift in the stoichiometry of trans-acting regulators induces nuclear reprogramming, providing the scientific underpinnings for induction of pluripotent stem cells (iPS) by transcription factor overexpression. Blau applied this discovery to stem cell biology and regenerative medicine. Capitalizing on the rapid, efficient, and synchronous reprogramming typical of heterokaryons, she showed that reprogramming entails a precise temporal sequence of gene expression. This finding enabled the identification of novel early regulators that proved crucial to the initiation of reprogramming to iPS and allowed analyses of the process of reprogramming through novel algorithms for next generation deep sequencing. She led the field by applying this new evidence of regenerative potential to novel approaches for treating muscle damaged by disease, injury, or aging. Her clever and illuminating experiments coupled with novel technological advances (section (4) below) – hallmarks of her research – led to the discovery that in lethal Duchenne Muscular Dystrophy (DMD), reduced telomere length leads to exhaustion of stem cells in skeletal muscle and mitochondrial failure in cardiac muscle. Her mouse model is the first to recapitulate the dilated cardiomyopathy that causes death in DMD, enabling tests of novel therapeutic interventions. She pioneered the use of muscle stem cells for clinical applications, developing methods for the prospective isolation, purification, and characterization of the muscle stem cells that reside in the tissue and are dedicated to skeletal muscle tissue regeneration. A groundbreaking advance was the development of a method to propagate the stem cells in culture using bioengineered microenvironments that mimic crucial muscle tissue properties, which showed that biophysical and biochemical mechanisms synergize to control stem cell self-renewal. She determined that with aging, two-thirds of muscle stem cells develop a cell-autonomous defect in muscle regeneration. A screen of compounds using an innovative non-invasive bioluminescence assay of muscle cell engraftment led to the discovery of a drug (SB202) that targets the intrinsic defect (p38α and p38β MAPK), leading to rejuvenation of the aged muscle stem cell population. Transplantation of treated aged stem cells into injured muscles of aged mice restored muscle strength to a level comparable to young. This work has broad therapeutic implications for treating muscle wasting due to localized trauma or atrophy following injury and immobilization due to bone fractures that plague the increasingly aged population. Blau has fundamentally altered our understanding of cell differentiation, profoundly impacting developmental biology, genetic reprogramming, stem cell research, and regenerative medicine.

Professional Experience
  • 1969 – 1975: Predoctoral Fellow, Department of Biology, Harvard University, Cambridge, MA (advisor: F.C. Kafatos)
  • 1975 – 1978: Postdoctoral Fellow, Division of Medical Genetics, Departments of Biochemistry and Biophysics, University of California, San Francisco, CA (advisor: C.J. Epstein)
  • 1978 – 1986: Assistant Professor, Department of Pharmacology, Stanford University School of Medicine, Stanford, CA
  • 1986 – 1991: Associate Professor, Department of Pharmacology, Stanford University School of Medicine, Stanford, CA
  • 1991 – 2002: Professor, Department of Molecular Pharmacology, Stanford University School of Medicine, Stanford, CA
  • 1997 – 2002: Chair, Department of Molecular Pharmacology (now Department of Chemical and Systems Biology), Stanford University School of Medicine, Stanford, CA
  • 1997 – present: Director, Gene Therapy Technology, Stanford University School of Medicine, Stanford, CA
  • 1999 – present: Donald E. and Delia B. Baxter Foundation Professor, Stanford University School of Medicine, Stanford, CA
  • 2002 – present: Director, Baxter Laboratory for Stem Cell Biology, Department of Microbiology and Immunology & Institute for Stem Cell Biology and Regenerative Medicine, Stanford University School of Medicine, Stanford, CA
 
MENTORS

Blau, Helen Margaret
Ph.D.
Duncan, James S
Ph.D.
Kishony, Roy
Ph.D.
Lin, Haifan
Ph.D.
Schoeler, Hans
Ph.D.
Spiegel, David
M.D.
Watt, Fiona M.
Ph.D., FRS, FMedSci