Research Background

​Genovese Lab

Dr. Pietro Genovese was recently a Project Leader at the San Raffaele Telethon Institute for Gene Therapy, having completed his post-doctoral fellowship in the laboratory of Molecular and Cell Biology of Gene Transfer with Professor Luigi Naldini in Italy. His current research focuses on gene-engineering technologies with a focus on hematopoietic stem cells and T cells. His future aim is to broaden his studies on pre-clinical development and to advance genetic engineering strategies with the aim to develop new treatment for human diseases, including cancer. At Dana-Farber/Boston Children's, Dr. Genovese joins the Gene Therapy Program in Pediatric Hematology/Oncology and the Program for Gene Therapy of Rare Diseases in the Department of Pediatrics (DOP). He has already had an impressively productive post-doctoral career with multiple publications, successful grant applications, and patents filed.

Publications

  1. Haploinsufficiency at the CX3CR1 locus of hematopoietic stem cells favors the appearance of microglia-like cells in the central nervous system of transplant recipients. Nat Commun. 2024 Nov 25; 15(1):10192. View Abstract
  2. Engineered packaging cell line for the enhanced production of baboon-enveloped retroviral vectors. Mol Ther Nucleic Acids. 2024 Dec 10; 35(4):102389. View Abstract
  3. Author Correction: IL-12 reprograms CAR-expressing natural killer T cells to long-lived Th1-polarized cells with potent antitumor activity. Nat Commun. 2024 Jun 18; 15(1):5182. View Abstract
  4. Enhancing prime editing in hematopoietic stem and progenitor cells by modulating nucleotide metabolism. Nat Biotechnol. 2024 May 28. View Abstract
  5. TIM-3, LAG-3, or 2B4 gene disruptions increase the anti-tumor response of engineered T cells. Front Immunol. 2024; 15:1315283. View Abstract
  6. Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency. Sci Transl Med. 2024 Feb 07; 16(733):eadh8162. View Abstract
  7. IL-12 reprograms CAR-expressing natural killer T cells to long-lived Th1-polarized cells with potent antitumor activity. Nat Commun. 2024 01 02; 15(1):89. View Abstract
  8. Epitope editing enables targeted immunotherapy of acute myeloid leukaemia. Nature. 2023 Sep; 621(7978):404-414. View Abstract
  9. Base Editing of Human Hematopoietic Stem Cells. Methods Mol Biol. 2023; 2606:43-62. View Abstract
  10. Real life hexavalent vaccination among children as a practical guide for public health professionals: Four years (from 2016 to 2019) of clinical practice in Sicily, Italy. Hum Vaccin Immunother. 2022 Nov 30; 18(6):2141998. View Abstract
  11. Therapeutic gene editing of T cells to correct CTLA-4 insufficiency. Sci Transl Med. 2022 10 26; 14(668):eabn5811. View Abstract
  12. Mesenchymal stromal cells improve the transplantation outcome of CRISPR-Cas9 gene-edited human HSPCs. Mol Ther. 2022 Oct 05; 30(10):3333. View Abstract
  13. Mesenchymal stromal cells improve the transplantation outcome of CRISPR-Cas9 gene-edited human HSPCs. Mol Ther. 2023 01 04; 31(1):230-248. View Abstract
  14. Editorial: Mutation-Specific Gene Editing for Blood Disorders. Front Genome Ed. 2021; 3:761771. View Abstract
  15. Retrieval of vector integration sites from cell-free DNA. Nat Med. 2021 08; 27(8):1458-1470. View Abstract
  16. BAR-Seq clonal tracking of gene-edited cells. Nat Protoc. 2021 06; 16(6):2991-3025. View Abstract
  17. Gene Editing of Hematopoietic Stem Cells: Hopes and Hurdles Toward Clinical Translation. Front Genome Ed. 2021; 3:618378. View Abstract
  18. Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper-IgM syndrome. EMBO Mol Med. 2021 03 05; 13(3):e13545. View Abstract
  19. Efficient gene editing of human long-term hematopoietic stem cells validated by clonal tracking. Nat Biotechnol. 2020 11; 38(11):1298-1308. View Abstract
  20. Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response. Cell Stem Cell. 2019 04 04; 24(4):551-565.e8. View Abstract
  21. Cyclosporine H Overcomes Innate Immune Restrictions to Improve Lentiviral Transduction and Gene Editing In Human Hematopoietic Stem Cells. Cell Stem Cell. 2018 12 06; 23(6):820-832.e9. View Abstract
  22. Therapeutic gene editing in CD34+ hematopoietic progenitors from Fanconi anemia patients. EMBO Mol Med. 2017 11; 9(11):1574-1588. View Abstract
  23. Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1. Sci Transl Med. 2017 Oct 11; 9(411). View Abstract
  24. NY-ESO-1 TCR single edited stem and central memory T cells to treat multiple myeloma without graft-versus-host disease. Blood. 2017 08 03; 130(5):606-618. View Abstract
  25. Cytokine-induced killer cells engineered with exogenous T-cell receptors directed against melanoma antigens: enhanced efficacy of effector cells endowed with a double mechanism of tumor recognition. Hum Gene Ther. 2015 Apr; 26(4):220-31. View Abstract
  26. Targeted genome editing in human repopulating haematopoietic stem cells. Nature. 2014 Jun 12; 510(7504):235-240. View Abstract
  27. Targeted gene therapy and cell reprogramming in Fanconi anemia. EMBO Mol Med. 2014 Jun; 6(6):835-48. View Abstract
  28. CD44v6-targeted T cells mediate potent antitumor effects against acute myeloid leukemia and multiple myeloma. Blood. 2013 Nov 14; 122(20):3461-72. View Abstract
  29. Editing T cell specificity towards leukemia by zinc finger nucleases and lentiviral gene transfer. Nat Med. 2012 May; 18(5):807-815. View Abstract
  30. Site-specific integration and tailoring of cassette design for sustainable gene transfer. Nat Methods. 2011 Aug 21; 8(10):861-9. View Abstract
  31. An unbiased genome-wide analysis of zinc-finger nuclease specificity. Nat Biotechnol. 2011 Aug 07; 29(9):816-23. View Abstract
  32. Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk. Hepatology. 2011 May; 53(5):1696-707. View Abstract
  33. Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nat Biotechnol. 2007 Nov; 25(11):1298-306. View Abstract

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