Serena Scala
Milan, 20132, Italy
Last Updated: 09/26/2026
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Found 33 publications
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
Molecular and phenotypic blueprint of human hematopoiesis links proliferation stress to stem cell aging.
Journal: The Journal of experimental medicine
Published: September 02, 2025
Evolution of hematopoietic stem cell potential from preterm to term neonates.
Journal: HemaSphere
Published: August 05, 2025
Development and characterization of a model of mucopolysaccharidosis type IVA for evaluating therapies targeting bone disease.
Journal: Disease models & mechanisms
Published: June 27, 2025
Mechanisms of hematopoietic clonal dominance in VEXAS syndrome.
Journal: Nature medicine
Published: July 08, 2024
Correction of osteopetrosis in the neonate oc/oc murine model after lentiviral vector gene therapy and non-genotoxic conditioning.
Journal: Frontiers in endocrinology
Published: June 17, 2024
Early bone marrow alterations in patients with adenosine deaminase 2 deficiency across disease phenotypes and severities.
Journal: The Journal of allergy and clinical immunology
Published: May 06, 2024
Transcriptomic analysis of BM-MSCs identified EGR1 as a transcription factor to fully exploit their therapeutic potential.
Journal: Biochimica et biophysica acta. Molecular cell research
Published: April 24, 2024
A GLB1 transgene with enhanced therapeutic potential for the preclinical development of ex-vivo gene therapy to treat mucopolysaccharidosis type IVB.
Journal: Molecular therapy. Methods & clinical development
Published: February 13, 2024
In vivo haemopoietic stem cell gene therapy enabled by postnatal trafficking.
Journal: Nature
Published: February 08, 2024
Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency.
Journal: Science translational medicine
Published: February 07, 2024
A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID.
Journal: Nature communications
Published: October 13, 2023
Last Updated: 09/26/2026