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Items: 11

1.

Liver-directed lentiviral gene therapy in a dog model of hemophilia B.

Cantore A, Ranzani M, Bartholomae CC, Volpin M, Valle PD, Sanvito F, Sergi LS, Gallina P, Benedicenti F, Bellinger D, Raymer R, Merricks E, Bellintani F, Martin S, Doglioni C, D'Angelo A, VandenDriessche T, Chuah MK, Schmidt M, Nichols T, Montini E, Naldini L.

Sci Transl Med. 2015 Mar 4;7(277):277ra28. doi: 10.1126/scitranslmed.aaa1405.

2.

miR-135a Inhibits Cancer Stem Cell-Driven Medulloblastoma Development by Directly Repressing Arhgef6 Expression.

Hemmesi K, Squadrito ML, Mestdagh P, Conti V, Cominelli M, Piras IS, Sergi LS, Piccinin S, Maestro R, Poliani PL, Speleman F, De Palma M, Galli R.

Stem Cells. 2015 May;33(5):1377-89. doi: 10.1002/stem.1958.

3.

Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosis.

Chiriaco M, Farinelli G, Capo V, Zonari E, Scaramuzza S, Di Matteo G, Sergi LS, Migliavacca M, Hernandez RJ, Bombelli F, Giorda E, Kajaste-Rudnitski A, Trono D, Grez M, Rossi P, Finocchi A, Naldini L, Gentner B, Aiuti A.

Mol Ther. 2014 Aug;22(8):1472-1483. doi: 10.1038/mt.2014.87. Epub 2014 May 29.

4.

Lentiviral vector-based insertional mutagenesis identifies genes associated with liver cancer.

Ranzani M, Cesana D, Bartholomae CC, Sanvito F, Pala M, Benedicenti F, Gallina P, Sergi LS, Merella S, Bulfone A, Doglioni C, von Kalle C, Kim YJ, Schmidt M, Tonon G, Naldini L, Montini E.

Nat Methods. 2013 Feb;10(2):155-61. doi: 10.1038/nmeth.2331. Epub 2013 Jan 13.

5.

Gene signatures associated with mouse postnatal hindbrain neural stem cells and medulloblastoma cancer stem cells identify novel molecular mediators and predict human medulloblastoma molecular classification.

Corno D, Pala M, Cominelli M, Cipelletti B, Leto K, Croci L, Barili V, Brandalise F, Melzi R, Di Gregorio A, Sergi LS, Politi LS, Piemonti L, Bulfone A, Rossi P, Rossi F, Consalez GG, Poliani PL, Galli R.

Cancer Discov. 2012 Jun;2(6):554-68. doi: 10.1158/2159-8290.CD-11-0199. Epub 2012 May 3. Erratum in: Cancer Discov. 2012 Sep;2(9):856. Daniela, Corno [corrected to Corno, Daniela].

6.

HIV-1-derived lentiviral vectors directly activate plasmacytoid dendritic cells, which in turn induce the maturation of myeloid dendritic cells.

Rossetti M, Gregori S, Hauben E, Brown BD, Sergi LS, Naldini L, Roncarolo MG.

Hum Gene Ther. 2011 Feb;22(2):177-88. doi: 10.1089/hum.2010.085. Epub 2011 Jan 14.

PMID:
20825284
7.

In vivo delivery of a microRNA-regulated transgene induces antigen-specific regulatory T cells and promotes immunologic tolerance.

Annoni A, Brown BD, Cantore A, Sergi LS, Naldini L, Roncarolo MG.

Blood. 2009 Dec 10;114(25):5152-61. doi: 10.1182/blood-2009-04-214569.

8.

Characterization of new arylsulfatase A gene mutations reinforces genotype-phenotype correlation in metachromatic leukodystrophy.

Cesani M, Capotondo A, Plati T, Sergi LS, Fumagalli F, Roncarolo MG, Naldini L, Comi G, Sessa M, Biffi A.

Hum Mutat. 2009 Oct;30(10):E936-45. doi: 10.1002/humu.21093.

PMID:
19606494
9.

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy.

Montini E, Cesana D, Schmidt M, Sanvito F, Bartholomae CC, Ranzani M, Benedicenti F, Sergi LS, Ambrosi A, Ponzoni M, Doglioni C, Di Serio C, von Kalle C, Naldini L.

J Clin Invest. 2009 Apr;119(4):964-75. doi: 10.1172/JCI37630. Epub 2009 Mar 23.

10.

A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice.

Brown BD, Cantore A, Annoni A, Sergi LS, Lombardo A, Della Valle P, D'Angelo A, Naldini L.

Blood. 2007 Dec 15;110(13):4144-52. Epub 2007 Aug 28.

11.

In vivo administration of lentiviral vectors triggers a type I interferon response that restricts hepatocyte gene transfer and promotes vector clearance.

Brown BD, Sitia G, Annoni A, Hauben E, Sergi LS, Zingale A, Roncarolo MG, Guidotti LG, Naldini L.

Blood. 2007 Apr 1;109(7):2797-805.

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