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Nature Communications|November 25, 2025
Microbiome modulation uncouples efficacy and toxicity induced by immune checkpoint blockade in mouse multiple myelomaLaura Lucia Cogrossi, Anna Policastro, Paola Zordan, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|March 5, 2009
Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical modelsFrancesco Marangoni, Marita Bosticardo, Sabine Charrier, et al.
EMBO Molecular Medicine|March 31, 2016
Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe-affected non-human primates by intracerebral lentiviral gene therapyVasco Meneghini, Annalisa Lattanzi, Luigi Tiradani, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|January 21, 2014
Uncovering and dissecting the genotoxicity of self-inactivating lentiviral vectors in vivoDaniela Cesana, Marco Ranzani, Monica Volpin, et al.
Oncotarget|July 14, 2016
Divergent in vitro/in vivo responses to drug treatments of highly aggressive NIH-Ras cancer cells: a PET imaging and metabolomics-mass-spectrometry studyDaniela Gaglio, Silvia Valtorta, Marilena Ripamonti, et al.
Molecular Therapy. Methods & Clinical Development|September 11, 2023
Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated in vitro and in vivoClaudia Asperti, Daniele Canarutto, Simona Porcellini, et al.
Science Translational Medicine|July 20, 2022
Targeted inducible delivery of immunoactivating cytokines reprograms glioblastoma microenvironment and inhibits growth in mouse modelsFilippo Birocchi, Melania Cusimano, Federico Rossari, et al.
Science Translational Medicine|October 13, 2017
Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1Giulia Schiroli, Samuele Ferrari, Anthony Conway, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|December 26, 2025
Tweaking lentiviral vector design balances efficacy and safety in liver-directed gene therapy for familial hypercholesterolemiaCesare Canepari, Michela Milani, Sara Degl'Innocenti, et al.
Science Translational Medicine|January 19, 2022
Disrupting N-glycan expression on tumor cells boosts chimeric antigen receptor T cell efficacy against solid malignanciesBeatrice Greco, Valeria Malacarne, Federica De Girardi, et al.
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