Fabrizio Benedicenti

Milan, 20132, Italy

Last Updated: 09/26/2026

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Found 35 publications

Gene therapy supports long-term reconstitution of patient hematopoietic stem cells in deficiency of adenosine deaminase 2.

Gene therapy supports long-term reconstitution of patient hematopoietic stem cells in deficiency of adenosine deaminase 2.

Journal: Molecular therapy. Methods & clinical development

Published: September 16, 2025

Nanoengineered 3D culture substrate enables superior persistence and polyclonal engraftment of genetically engineered hematopoietic stem cells.

Nanoengineered 3D culture substrate enables superior persistence and polyclonal engraftment of genetically engineered hematopoietic stem cells.

Journal: Cell stem cell

Published: June 18, 2025

Liver-directed lentiviral gene therapy confers durable hepatic and systemic amelioration of methylmalonic acidemia in mice.

Liver-directed lentiviral gene therapy confers durable hepatic and systemic amelioration of methylmalonic acidemia in mice.

Journal: Journal of hepatology

Published: March 28, 2025

Donor-derived CARCIK-CD19 cells engineered with Sleeping Beauty transposon in acute lymphoblastic leukemia relapsed after allogeneic transplantation.

Donor-derived CARCIK-CD19 cells engineered with Sleeping Beauty transposon in acute lymphoblastic leukemia relapsed after allogeneic transplantation.

Journal: Blood cancer journal

Published: January 08, 2025

A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID.

A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID.

Journal: Nature communications

Published: October 13, 2023

Circulating hematopoietic stem/progenitor cell subsets contribute to human hematopoietic homeostasis.

Circulating hematopoietic stem/progenitor cell subsets contribute to human hematopoietic homeostasis.

Journal: Blood

Published: September 22, 2023

Removal of innate immune barriers allows efficient transduction of quiescent human hematopoietic stem cells.

Removal of innate immune barriers allows efficient transduction of quiescent human hematopoietic stem cells.

Journal: Molecular therapy : the journal of the American Society of Gene Therapy

Published: May 23, 2023

InCliniGene enables high-throughput and comprehensive in vivo clonal tracking toward clinical genomics data integration.

InCliniGene enables high-throughput and comprehensive in vivo clonal tracking toward clinical genomics data integration.

Journal: Database : the journal of biological databases and curation

Published: November 10, 2022

ISAnalytics enables longitudinal and high-throughput clonal tracking studies in hematopoietic stem cell gene therapy applications.

ISAnalytics enables longitudinal and high-throughput clonal tracking studies in hematopoietic stem cell gene therapy applications.

Journal: Briefings in bioinformatics

Published: September 27, 2022

Long-term lineage commitment in haematopoietic stem cell gene therapy.

Long-term lineage commitment in haematopoietic stem cell gene therapy.

Journal: Nature

Published: September 16, 2022

Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy.

Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy.

Journal: Nature communications

Published: August 08, 2022

Intrathymic AAV delivery results in therapeutic site-specific integration at TCR loci in mice.

Intrathymic AAV delivery results in therapeutic site-specific integration at TCR loci in mice.

Journal: Blood

Published: June 10, 2022

Showing 1-12 of 35

Last Updated: 09/26/2026