Oncology Division

Mark S. Sands, PhD

Department of Medicine
Oncology Division
Stem Cell Biology
Department of Genetics

Research Interests

  • Lysosomal storage diseases
  • Neurodegenerative disorders
  • Gene therapy
  • Enzyme replacement
  • Hematopoiesis
  • Bone marrow transplantation


  • 314-362-5494 (office)
  • 314-362-5496 (lab)
  • 314-747-5770 (fax)
  • Room 5509, 4444 Forest Park (office)
  • Room 5510, 4444 Forest Park (lab)
  • Division of Oncology
    Section of Stem Cell Biology
    Washington University Medical School
    660 South Euclid Avenue - Box 8007
    St. Louis, MO 63110

Invited Reviews & Book Chapters

  • Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights From Animal Models
    Chandler RJ, Sands MS, Venditti CP
    Hum Gene Ther 2017 Apr;28(4):314-322
  • Treatment for Krabbe's Disease: Finding the Combination
    Mikulka CR, Sands MS
    J Neurosci Res 2016 Nov;94(11):1126-37
  • Overcoming the Next Barriers to Successful Therapy
    Cohen IJ, Baris H, Mistry PK, Sands MS
    Pediatr Endocrinol Rev 2016 Jun;13 Suppl 1:629
  • Insights Into the Pathogenesis and Treatment of Krabbe Disease
    Bongarzone ER, Escolar ML, Gray SJ, Kafri T, Vite CH, Sands MS
    Pediatr Endocrinol Rev 2016 Jun;13 Suppl 1:689-96
  • Mucopolysaccharidosis Type VII: A Powerful Experimental System and Therapeutic Challenge
    Sands MS
    Pediatr Endocrinol Rev 2014 Sep;12 Suppl 1:159-65
  • Treatment for LSDs: Real Options for Several Diseases. Forward
    Cohen IJ, Baris HN, Mistry PK, Sands MS
    Pediatr Endocrinol Rev 2014 Sep;12 Suppl 1:71
  • A Hitchhiker's Guide to the Blood-Brain Barrier: In Trans Delivery of a Therapeutic Enzyme
    Sands MS
    Mol Ther 2014 Mar;22(3):483-4
  • Pathogenesis and Therapies for Infantile Neuronal Ceroid Lipofuscinosis (Infantile CLN1 Disease)
    Hawkins-Salsbury JA, Cooper JD, Sands MS
    Biochim Biophys Acta 2013 Nov;1832(11):1906-9
  • Considerations for the Treatment of Infantile Neuronal Ceroid Lipofuscinosis (Infantile Batten Disease)
    Sands MS
    J Child Neurol 2013 Sep;28(9):1151-8
  • Farber Disease: Understanding a Fatal Childhood Disorder and Dissecting Ceramide Biology
    Sands MS
    EMBO Mol Med 2013 Jun;5(6):799-801
  • AAV-Mediated Liver-Directed Gene Therapy
    Sands MS
    In: Methods in Molecular Biology (RO Snyder, ed), Humana Press Inc., Totowa, NJ 807:141; 2011
  • Combination Therapies for Lysosomal Storage Disease: Is the Whole Greater Than the Sum of Its Parts?
    Hawkins-Salsbury JA, Reddy AS, Sands MS
    Hum Mol Genet 2011 Apr 15;20(R1):R54-60
  • Promising CNS-Directed Enzyme Replacement Therapy for Lysosomal Storage Diseases
    Macauley SL, Sands MS
    Exp Neurol 2009 Jul;218(1):5-8
  • CNS-Directed Gene Therapy for Lysosomal Storage Diseases
    Sands MS, Haskins ME
    Acta Paediatr Suppl 2008 Apr;97(457):22-7
  • Gene Therapy for Lysosomal Storage Diseases
    Sands MS, Davidson BL
    Mol Ther 2006 May;13(5):839-49
  • Pre-Clinical Basis for Gene Therapy Studies
    Sands MS, Nolta JA, Bauer G, Ferkol T
    In: Translational and Experimental Medicine (Schuster D, Powers W, eds), Lippincott Publishing; 2005
  • Gene Therapy for Lysosomal Storage Diseases Affecting the Brain
    Sands MS
    In: Lysosomal Storage Diseases Affecting the Central Nervous System (Platt F, Walkley SU, eds), Oxford University Press, Oxford, UK; 2004
  • Safety of Adeno-Associated Virus Gene Therapy Vectors: A Current Evaluation
    Monahan PE, Jooss K, Sands MS
    Expert Opin Drug Saf 2002 May;1(1):79-91
  • Preventing Sensory Loss in a Mouse Model of Lysosomal Storage Disease
    Ohlemiller KK, Vogler CA, Daly TM, Sands MS
    In: Handbook of mouse auditory research: from behavior to molecular biology (JF Willot, ed), CRC Press, New York; 2001
  • Gene Therapy for Lysosomal Storage Diseases
    Daly TM, Sands MS
    Expert Opin Investig Drugs 1998 Oct;7(10):1673-82
  • Murine Mucopolysaccharidosis Type VII: A Model System for Somatic Gene Therapy of the Central Nervous System
    Wolfe JH, Sands MS
    In: Gene Transfer into Neurones, Towards Gene Therapy of Neurological Disorders (P Lowenstein, L Enquist, eds), J Wiley & Sons Ltd, Essex, England, p 263; 1996
  • Domains of the 5S RNA Gene Control Region
    Bogenhagen DF, Sands MS, McConkey GA
    16th Annual Steenbock Symposium (Reznikoff W et al, eds), Madison, Wisconsin, p 219; 1986
  • Maximizing Hepatic Contrast Enhancement With a Particulate Contrast Agent in Computed Tomography
    Violante MR, Fischer HW, Mare KO, Boxmann HB, Owunwanne A, Zack AG, Harmon P, Case K, Sands M
    Contrast Media in Computed Tomography, International Workshop, Berlin, January, 1981