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Showing 1–8 of 8 results
Advanced filters: Author: Stefania Cesana Clear advanced filters

  • Fifteen years’ follow-up of clinical development and real-world data from 43 patients show that gammaretroviral gene therapy for adenosine deaminase deficiency has a positive long-term efficacy profile, warranting continued safety monitoring of patients receiving gene therapy.

    • Maddalena Migliavacca
    • Federica Barzaghi
    • Maria Pia Cicalese
    Research
    Nature Medicine
    Volume: 30, P: 488-497

  • Haematopoietic stem cell (HSC) clonogenic activity, lineage output, lineage commitment and somatic mutation rates are influenced by the underlying disease, patient age, extent of genetic defect correction and hematopoietic stress imposed by the inherited disease, suggesting HSC adaptation.

    • Andrea Calabria
    • Giulio Spinozzi
    • Eugenio Montini
    ResearchOpen Access
    Nature
    Volume: 636, P: 162-171

  • Leukaemia development has been reported as an associated risk of haematopoietic stem cell gene therapy (HSPC-GT) using retroviral vectors in different diseases. Here, the authors show a case of T-cell acute lymphoid leukaemia in a patient with Adenosine Deaminase-deficient Severe Combined Immunodeficiency (ADA-SCID) treated with retroviral gene therapy.

    • Daniela Cesana
    • Maria Pia Cicalese
    • Alessandro Aiuti
    ResearchOpen Access
    Nature Communications
    Volume: 15, P: 1-18

  • A genome-wide association study and Metabochip meta-analysis of body mass index (BMI) detects 97 BMI-associated loci, of which 56 were novel, and many loci have effects on other metabolic phenotypes; pathway analyses implicate the central nervous system in obesity susceptibility and new pathways such as those related to synaptic function, energy metabolism, lipid biology and adipogenesis.

    • Adam E. Locke
    • Bratati Kahali
    • Elizabeth K. Speliotes
    Research
    Nature
    Volume: 518, P: 197-206

  • Cristen Willer and colleagues report genome-wide association analyses for blood lipid levels in 188,578 individuals. They identify 62 loci newly associated with blood lipid levels, refine the association signals at 12 loci and examine associations with cardiovascular and metabolic traits.

    • Cristen J Willer
    • Ellen M Schmidt
    • Gonçalo R Abecasis
    Research
    Nature Genetics
    Volume: 45, P: 1274-1283