R. Scott McIvor

University of Minnesota — United States

Biochemistry, Genetics and Molecular Biology · Life Sciences

45h-index6.9kcitations238works5.32yr avg
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Research Topics

Virus-based gene therapy research(112), Lysosomal Storage Disorders Research(58), CRISPR and Genetic Engineering(57), RNA Interference and Gene Delivery(54), CAR-T cell therapy research(36)

Publications238 total

Targeting the intracellular immune checkpoint CISH with CRISPR-Cas9-edited T cells in patients with metastatic colorectal cancer: a first-in-human, single-centre, phase 1 trial
The Lancet Oncology·2025· 32 cited
Abstract CT269: First-in-human trial in patients with metastatic colorectal cancer using CRISPR-engineered tumor infiltrating lymphocytes in which the intracellular immune checkpoint <i>CISH</i> is inhibited
Cancer Research·2025· 1 cited
Intrathecal or intravenous AAV9-IDUA/RGX-111 at minimal effective dose prevents cardiac, skeletal and neurologic manifestations of murine MPS I
Molecular Therapy — Methods & Clinical Development·2024· 1 citedOpen Access
Advancing gene targeting for primary immune deficiencies: Adenine base editing of the human IL2RG locus for correction of SCID-X1
Molecular Therapy·2024Open Access
Adeno-associated virus-mediated gene transfer of arginine decarboxylase to the central nervous system prevents opioid analgesic tolerance
Frontiers in Pain Research·2024· 3 citedOpen Access
Identification of inflammatory cells in dilated ascending aortas of IDUA-deficient (MPS I) mice
Molecular Genetics and Metabolism·2024
First-in-human clinical trial of genetically engineered B cells: Application to the treatment of mucopolysaccharidosis type I
Molecular Genetics and Metabolism·2024Open Access
Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice
Molecular Therapy — Methods & Clinical Development·2024· 5 citedOpen Access
Cas9-induced targeted integration of large DNA payloads in primary human T cells via homology-mediated end-joining DNA repair
Nature Biomedical Engineering·2023· 20 citedOpen Access
AAV-mediated gene transfer to colon-innervating primary afferent neurons
Frontiers in Pain Research·2023· 1 citedOpen Access
Generation and characterization of an immunodeficient mouse model of mucopolysaccharidosis type II
Molecular Genetics and Metabolism·2023· 2 citedOpen Access
Long-term reversal of chronic pain behavior in rodents through elevation of spinal agmatine
Molecular Therapy·2023· 13 citedOpen Access
Non-invasive intravenous administration of AAV9 transducing iduronate sulfatase leads to global metabolic correction and prevention of neurologic deficits in a mouse model of Hunter syndrome
Molecular Genetics and Metabolism Reports·2023· 6 citedOpen Access
Lentiviral Gene Therapy for Artemis-Deficient SCID
New England Journal of Medicine·2022· 68 citedOpen Access
Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS After the Development of Cognitive Dysfunction
Human Gene Therapy·2022· 8 citedOpen Access
Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of <i>Ex Vivo</i> Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells
Human Gene Therapy·2022· 14 citedOpen Access
Treatment of cerebral adrenoleukodystrophy: allogeneic transplantation and lentiviral gene therapy
Expert Opinion on Biological Therapy·2022· 59 citedOpen Access
Internal checkpoint regulates T cell neoantigen reactivity and susceptibility to PD1 blockade
Med·2022· 69 citedOpen Access
Correction of Fanconi Anemia Mutations Using Digital Genome Engineering
International Journal of Molecular Sciences·2022· 11 citedOpen Access
Contribution of the innate and adaptive immune systems to aortic dilation in murine mucopolysaccharidosis type I
Molecular Genetics and Metabolism·2022· 6 citedOpen Access
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Frequent Co-authors

Perry B. Hackett(11), Lalitha R. Belur(10), Walter C. Low(8), Andrew Wilber(7), Kelly M. Podetz-Pedersen(7), Joel L. Frandsen(7), Chester B. Whitley(7), Paul J. Orchard(7), David A. Largaespada(6), Elena L. Aronovich(6), Jakub Tolar(5), Bruce R. Blazar(5), Jason Bell(5), Stephen C. Ekker(4), Maureen Riedl(4), Kelley F. Kitto(4), Carolyn A. Fairbanks(4), Lucy Vulchanova(4), Roland Günther(4), Brenda Koniar(4)