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Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis.
Chiriaco, Maria; Farinelli, Giada; Capo, Valentina; Zonari, Erika; Scaramuzza, Samantha; Di Matteo, Gigliola; Sergi, Lucia Sergi; Migliavacca, Maddalena; Hernandez, Raisa Jofra; Bombelli, Ferdinando; Giorda, Ezio; Kajaste-Rudnitski, Anna; Trono, Didier; Grez, Manuel; Rossi, Paolo; Finocchi, Andrea; Naldini, Luigi; Gentner, Bernhard; Aiuti, Alessandro.
Affiliation
  • Chiriaco M; Department of Pediatrics, Children's Hospital Bambino Gesù and University of Rome Tor Vergata School of Medicine, Rome, Italy.
  • Farinelli G; San Raffaele Telethon Institute for Gene Therapy (TIGET), Scientific Institute HS Raffaele, Milan, Italy.
  • Capo V; Department of Pediatrics, Children's Hospital Bambino Gesù and University of Rome Tor Vergata School of Medicine, Rome, Italy.
  • Zonari E; San Raffaele Telethon Institute for Gene Therapy (TIGET), Scientific Institute HS Raffaele, Milan, Italy.
  • Scaramuzza S; San Raffaele Telethon Institute for Gene Therapy (TIGET), Scientific Institute HS Raffaele, Milan, Italy.
  • Di Matteo G; Department of Pediatrics, Children's Hospital Bambino Gesù and University of Rome Tor Vergata School of Medicine, Rome, Italy.
  • Sergi LS; San Raffaele Telethon Institute for Gene Therapy (TIGET), Scientific Institute HS Raffaele, Milan, Italy.
  • Migliavacca M; San Raffaele Telethon Institute for Gene Therapy (TIGET), Scientific Institute HS Raffaele, Milan, Italy.
  • Hernandez RJ; San Raffaele Telethon Institute for Gene Therapy (TIGET), Scientific Institute HS Raffaele, Milan, Italy.
  • Bombelli F; Scientific Institute HS Raffaele, Milan, Italy.
  • Giorda E; Laboratory of Flow Cytometry and B Cell Development, Children's Hospital Bambino Gesù, Rome, Italy.
  • Kajaste-Rudnitski A; San Raffaele Telethon Institute for Gene Therapy (TIGET), Scientific Institute HS Raffaele, Milan, Italy.
  • Trono D; École Polytechnique Fédérale de Lausanne, Lausanne, Switzerland.
  • Grez M; Georg-Speyer Haus, Frankfurt, Germany.
  • Rossi P; Department of Pediatrics, Children's Hospital Bambino Gesù and University of Rome Tor Vergata School of Medicine, Rome, Italy.
  • Finocchi A; Department of Pediatrics, Children's Hospital Bambino Gesù and University of Rome Tor Vergata School of Medicine, Rome, Italy.
  • Naldini L; San Raffaele Telethon Institute for Gene Therapy (TIGET), Scientific Institute HS Raffaele, Milan, Italy; "Vita-Salute" S. Raffaele University, Milan, Italy.
  • Gentner B; San Raffaele Telethon Institute for Gene Therapy (TIGET), Scientific Institute HS Raffaele, Milan, Italy.
  • Aiuti A; Department of Pediatrics, Children's Hospital Bambino Gesù and University of Rome Tor Vergata School of Medicine, Rome, Italy; San Raffaele Telethon Institute for Gene Therapy (TIGET), Scientific Institute HS Raffaele, Milan, Italy. Electronic address: aiuti@med.uniroma2.it.
Mol Ther ; 22(8): 1472-1483, 2014 Aug.
Article in En | MEDLINE | ID: mdl-24869932

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Hematopoietic Stem Cells / Membrane Glycoproteins / Genetic Therapy / Hematopoietic Stem Cell Transplantation / NADPH Oxidases / MicroRNAs / Granulomatous Disease, Chronic Limits: Animals / Humans Language: En Journal: Mol Ther Journal subject: BIOLOGIA MOLECULAR / TERAPEUTICA Year: 2014 Document type: Article Affiliation country: Country of publication:

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Hematopoietic Stem Cells / Membrane Glycoproteins / Genetic Therapy / Hematopoietic Stem Cell Transplantation / NADPH Oxidases / MicroRNAs / Granulomatous Disease, Chronic Limits: Animals / Humans Language: En Journal: Mol Ther Journal subject: BIOLOGIA MOLECULAR / TERAPEUTICA Year: 2014 Document type: Article Affiliation country: Country of publication: