R. Scott McIvor

About

R. Scott McIvor, PhD, is a professor affiliated with the Genetics, Cell Biology & Development department and the Stem Cell Institute at the University of Minnesota Twin Cities. His laboratory's general research interest is in genetic therapy for inherited diseases and cancer. His specific research activities include the use of recombinant lentiviral vectors for gene transfer into hematopoietic stem cells to treat cancer and inherited disorders such as immunodeficiency diseases and lysosomal storage diseases. He also works on the use of adeno-associated virus for treating storage diseases and as a vector for gene transfer into the central nervous system for neurological disorders. Additionally, his research involves the adaptation of the vertebrate transposon 'Sleeping Beauty' in developing non-viral genetic therapies and chromosomal site-directed correction of mutations in hematopoietic stem cells and other tissues for treating immunodeficiencies and inherited disorders. Recent highlights of his work include restoring immunity in infants with severe combined immunodeficiency (SCID), supporting the first human clinical trial of in vivo gene editing, and advancing preclinical work for clinical trials of new genetic therapies for mucopolysaccharidoses MPS I and MPS II.

Research topics

• Medicine
• Biology
• Internal medicine
• Genetics
• Pathology
• Molecular biology
• Cancer research
• Organic chemistry
• Biochemistry
• Immunology

Selected publications

• Mucopolysaccharidosis Type I: A Review of the Natural History and Molecular Pathology

  Cells · 2020 · 98 citations

  Senior authorCorresponding

  Mucopolysaccharidosis type I (MPS I) is a rare autosomal recessive inherited disease, caused by deficiency of the enzyme α-L-iduronidase, resulting in accumulation of the glycosaminoglycans (GAGs) dermatan and heparan sulfate in organs and tissues. If untreated, patients with the severe phenotype die within the first decade of life. Early diagnosis is crucial to prevent the development of fatal disease manifestations, prominently cardiac and respiratory disease, as well as cognitive impairment.…

  DOI

• Mucopolysaccharidosis Type I: Current Treatments, Limitations, and Prospects for Improvement

  Biomolecules · 2021 · 85 citations

  Senior authorCorresponding

  Mucopolysaccharidosis type I (MPS I) is a lysosomal disease, caused by a deficiency of the enzyme alpha-L-iduronidase (IDUA). IDUA catalyzes the degradation of the glycosaminoglycans dermatan and heparan sulfate (DS and HS, respectively). Lack of the enzyme leads to pathologic accumulation of undegraded HS and DS with subsequent disease manifestations in multiple organs. The disease can be divided into severe (Hurler syndrome) and attenuated (Hurler-Scheie, Scheie) forms. Currently approved trea…

  DOI

• Internal checkpoint regulates T cell neoantigen reactivity and susceptibility to PD1 blockade

  Med · 2022-08-24 · 74 citations

  articleOpen access

  BACKGROUND: Adoptive transfer of tumor-infiltrating lymphocytes (TIL) fails to consistently elicit tumor rejection. Manipulation of intrinsic factors that inhibit T cell effector function and neoantigen recognition may therefore improve TIL therapy outcomes. We previously identified the cytokine-induced SH2 protein (CISH) as a key regulator of T cell functional avidity in mice. Here, we investigate the mechanistic role of CISH in regulating human T cell effector function in solid tumors and demo…

  PublisherDOI

• Lentiviral Gene Therapy for Artemis-Deficient SCID

  New England Journal of Medicine · 2022-12-21 · 72 citations

  articleOpen access

  BACKGROUND: , which encodes Artemis, cause Artemis-deficient severe combined immunodeficiency (ART-SCID), which is poorly responsive to allogeneic hematopoietic-cell transplantation. METHODS: , in 10 infants with newly diagnosed ART-SCID. We followed them for a median of 31.2 months. RESULTS: Marrow harvest, busulfan conditioning, and lentiviral-transduced CD34+ cell infusion produced the expected grade 3 or 4 adverse events. All the procedures met prespecified criteria for feasibility at 42 day…

  PublisherOA PDFDOI

• Treatment of cerebral adrenoleukodystrophy: allogeneic transplantation and lentiviral gene therapy

  Expert Opinion on Biological Therapy · 2022-09-02 · 63 citations

  reviewOpen access

  INTRODUCTION: gene. By adulthood, approximately 40% of the patients develop cerebral ALD, a severe, neuroinflammatory condition that is generally progressive and fatal without intervention. AREAS COVERED: Historically, only allogeneic hematopoietic stem cell transplantation (HSCT) has been shown to halt progression of cerebral ALD, with superior outcomes obtained when HSCT is performed early in the disease process. More recently, a lentiviral-based gene therapy approach has been investigated as…

  PublisherOA PDFDOI

Recent grants

• Immune Monitoring Core

  NIH · $82.8M · 1997–2028

• NIH Grant R01AI063340

  NIH · $1.4M · 2011

• NIH Grant R01CA060803

  NIH · $3.7M · 2011

Frequent coauthors

• Perry B. Hackett

  University of Minnesota

  61 shared

• Lalitha R. Belur

  University of Minnesota

  59 shared

• Kelly M. Podetz-Pedersen

  University of Minnesota

  52 shared

• Bruce R. Blazar

  University of Minnesota

  47 shared

• Jakub Tolar

  42 shared

• Chester B. Whitley

  41 shared

• Walter C. Low

  University of Minnesota

  39 shared

• Andrew Wilber

  Southern Illinois University School of Medicine

  37 shared

Education

• Ph.D., Microbiology

  University of Minnesota

  1982

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