Affiliation: Federico II University
- Identification of molecular defects in Italian Sanfilippo A patients including 13 novel mutationsP Di Natale
Dipartimento di Biochimica e Biotecnologie Mediche, Medical School, University of Naples Federico II, Italy
Hum Mutat 11:313-20. 1998..Interestingly, all six patients from Sardinia present this mutation, and five of them are homozygous for this change, suggesting that these subjects may have been derived from a common founder...
- Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector deliveryAngelo Lombardo
San Raffaele Telethon Institute for Gene Therapy, Via Olgettina, 58, 20132 Milan, Italy
Nat Biotechnol 25:1298-306. 2007....
- Heparan N-sulfatase: in vitro mutagenesis of potential N-glycosylation sitesP Di Natale
Department of Biochemistry and Medical Biotechnologies, University of Naples Federico II, Naples, Italy
Biochem Biophys Res Commun 280:1251-7. 2001..These studies confirm that the five glycosylation sites of heparan N-sulfatase are all functional and show that Asn 41 and Asn 151 have a role in protein folding and/or stability...
- In vitro gene therapy of mucopolysaccharidosis type I by lentiviral vectorsPaola Di Natale
Department of Biochemistry and Medical Biotechnologies, University of Naples Federico II, Italy
Eur J Biochem 269:2764-71. 2002..These results suggest that the lentiviral vector may be used for the delivery and expression of the IDUA gene to cells in vivo for treatment of MPS I...
- Gene therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vectorCarmela Di Domenico
Department of Biochemistry and Medical Biotechnologies, University of Naples Federico II, 80131 Naples, Italy
Hum Gene Ther 16:81-90. 2005..In conclusion, our results show the promising potential and the limitations of lentiviral-IDUA vector-mediated gene therapy in an in vivo model...
- Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in miceAntonia Follenzi
Laboratory of Gene Transfer and Therapy, Institute for Cancer Research and Treatment IRCC, University of Torino, Torino, Italy
Blood 103:3700-9. 2004..These results prompt further studies in relevant animal models to explore the potential of in vivo LV administration for the gene therapy of hemophilias and other liver-based diseases...
- The immune response to lentiviral-delivered transgene is modulated in vivo by transgene-expressing antigen-presenting cells but not by CD4+CD25+ regulatory T cellsAndrea Annoni
San Raffaele Telethon Institute for Gene Therapy HSR TIGET, Milan, Italy
Blood 110:1788-96. 2007..These data indicate that antitransgene immune response can be modulated by transgene-expressing APCs possibly through deletion of effector T cells...
- A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B miceBrian D Brown
San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy
Blood 110:4144-52. 2007..This work, which is among the first applications to exploit the microRNA regulatory pathway, provides the basis for a promising new therapy for the treatment of hemophilia B...
- Lentiviral gene transfer and ex vivo expansion of human primitive stem cells capable of primary, secondary, and tertiary multilineage repopulation in NOD/SCID mice. Nonobese diabetic/severe combined immunodeficientWanda Piacibello
Department of Oncological Sciences, University of Torino Medical School, Torino, Italy
Blood 100:4391-400. 2002..8% +/- 5.9% of human cells were GFP(+), and human engraftment was multilineage. These results show that lentiviral vectors efficiently transduce HSCs, which can undergo expansion and maintain proliferation and self-renewal ability...