L
Luigi Naldini
Researcher at Vita-Salute San Raffaele University
Publications - 370
Citations - 59320
Luigi Naldini is an academic researcher from Vita-Salute San Raffaele University. The author has contributed to research in topics: Genetic enhancement & Viral vector. The author has an hindex of 108, co-authored 345 publications receiving 55080 citations. Previous affiliations of Luigi Naldini include Università telematica San Raffaele & Sangamo BioSciences.
Papers
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Book ChapterDOI
Generation of HIV-1 derived lentiviral vectors.
Antonia Follenzi,Luigi Naldini +1 more
Journal ArticleDOI
In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice
Antonella Consiglio,Angelo Quattrini,Sabata Martino,Jean Charles Bensadoun,D. Dolcetta,Alessandra Trojani,Giuliana Benaglia,Sergio Marchesini,Vincenzo Cestari,Alberto Oliverio,Claudio Bordignon,Luigi Naldini +11 more
TL;DR: It is shown that selective degeneration of hippocampal neurons is a central step in disease pathogenesis, and evidence that in vivo transfer of ARSA by lentiviral vectors reverts the disease phenotype in all investigated areas is provided.
Journal ArticleDOI
Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response
Giulia Schiroli,Anastasia Conti,Samuele Ferrari,Lucrezia della Volpe,Aurelien Jacob,Luisa Albano,Stefano Beretta,Stefano Beretta,Andrea Calabria,Valentina Vavassori,Patrizia Gasparini,Eralda Salataj,Eralda Salataj,Eralda Salataj,Delphine Ndiaye-Lobry,Delphine Ndiaye-Lobry,Delphine Ndiaye-Lobry,Chiara Brombin,Julie Chaumeil,Julie Chaumeil,Julie Chaumeil,Eugenio Montini,Ivan Merelli,Pietro Genovese,Luigi Naldini,Raffaella Di Micco +25 more
TL;DR: Molecular and functional evidence for feasible and seamless gene editing in HSPCs is provided and p53-mediated DDR pathway activation was the predominant response to even single-nuclease-induced DSBs across all HSPC subtypes analyzed.
Journal ArticleDOI
Identification of Hematopoietic Stem Cell–Specific miRNAs Enables Gene Therapy of Globoid Cell Leukodystrophy
Bernhard Gentner,Ilaria Visigalli,Hidefumi Hiramatsu,Eric R. Lechman,Silvia Ungari,Alice Giustacchini,Giulia Schira,Mario Amendola,Angelo Quattrini,Sabata Martino,Aldo Orlacchio,John E. Dick,Alessandra Biffi,Luigi Naldini +13 more
TL;DR: Hematopoietic stem cell–specific microRNAs allow regulation of therapeutic transgene expression and enable effective gene therapy of globoid cell leukodystrophy and provide a basis for improvements in HSPC-mediated gene therapy and may offer patients a new therapeutic option that resembles a scratch more than a chop.
Journal ArticleDOI
In vivo administration of lentiviral vectors triggers a type I interferon response that restricts hepatocyte gene transfer and promotes vector clearance
Brian D. Brown,Giovanni Sitia,Andrea Annoni,Ehud Hauben,Lucia Sergi Sergi,Anna Zingale,Maria Grazia Roncarolo,Luca G. Guidotti,Luigi Naldini +8 more
TL;DR: It is shown that administration of LVs to mice triggers a rapid and transient IFNalphabeta response, and because disabling a single component of the innate/immune network was sufficient to establish persistent xenoantigen expression, the results raise the hope that the immunologic barriers to gene therapy are less insurmountable than expected.