Format
Sort by
Items per page

Send to

Choose Destination

Search results

Items: 1 to 50 of 178

1.

Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency.

Poletti V, Charrier S, Corre G, Gjata B, Vignaud A, Zhang F, Rothe M, Schambach A, Gaspar HB, Thrasher AJ, Mavilio F.

Mol Ther Methods Clin Dev. 2018 Mar 10;9:257-269. doi: 10.1016/j.omtm.2018.03.002. eCollection 2018 Jun 15.

2.

The Pharmacology of Gene and Cell Therapy.

Conlon TJ, Mavilio F.

Mol Ther Methods Clin Dev. 2018 Mar 15;8:181-182. doi: 10.1016/j.omtm.2017.12.007. eCollection 2018 Mar 16. No abstract available.

3.

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus.

Antoniani C, Meneghini V, Lattanzi A, Felix T, Romano O, Magrin E, Weber L, Pavani G, El Hoss S, Kurita R, Nakamura Y, Cradick TJ, Lundberg AS, Porteus M, Amendola M, El Nemer W, Cavazzana M, Mavilio F, Miccio A.

Blood. 2018 Apr 26;131(17):1960-1973. doi: 10.1182/blood-2017-10-811505. Epub 2018 Mar 8.

PMID:
29519807
4.

Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors.

Holstein M, Mesa-Nuñez C, Miskey C, Almarza E, Poletti V, Schmeer M, Grueso E, Ordóñez Flores JC, Kobelt D, Walther W, Aneja MK, Geiger J, Bonig HB, Izsvák Z, Schleef M, Rudolph C, Mavilio F, Bueren JA, Guenechea G, Ivics Z.

Mol Ther. 2018 Apr 4;26(4):1137-1153. doi: 10.1016/j.ymthe.2018.01.012. Epub 2018 Jan 31.

5.

Gene transfer into hematopoietic stem cells reduces HLH manifestations in a murine model of Munc13-4 deficiency.

Soheili T, Durand A, Sepulveda FE, Rivière J, Lagresle-Peyrou C, Sadek H, de Saint Basile G, Martin S, Mavilio F, Cavazzana M, André-Schmutz I.

Blood Adv. 2017 Dec 21;1(27):2781-2789. doi: 10.1182/bloodadvances.2017012088. eCollection 2017 Dec 26.

6.

Interactions between Retroviruses and the Host Cell Genome.

Poletti V, Mavilio F.

Mol Ther Methods Clin Dev. 2017 Oct 5;8:31-41. doi: 10.1016/j.omtm.2017.10.001. eCollection 2018 Mar 16. Review.

7.

Gene Therapy Approaches to Hemoglobinopathies.

Ferrari G, Cavazzana M, Mavilio F.

Hematol Oncol Clin North Am. 2017 Oct;31(5):835-852. doi: 10.1016/j.hoc.2017.06.010. Review.

PMID:
28895851
8.

Evaluation of tolerance to lentiviral LV-RPE65 gene therapy vector after subretinal delivery in non-human primates.

Matet A, Kostic C, Bemelmans AP, Moulin A, Rosolen SG, Martin S, Mavilio F, Amirjanians V, Stieger K, Lorenz B, Behar-Cohen F, Arsenijevic Y.

Transl Res. 2017 Oct;188:40-57.e4. doi: 10.1016/j.trsl.2017.06.012. Epub 2017 Jul 8.

9.

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy.

Le Guiner C, Servais L, Montus M, Larcher T, Fraysse B, Moullec S, Allais M, François V, Dutilleul M, Malerba A, Koo T, Thibaut JL, Matot B, Devaux M, Le Duff J, Deschamps JY, Barthelemy I, Blot S, Testault I, Wahbi K, Ederhy S, Martin S, Veron P, Georger C, Athanasopoulos T, Masurier C, Mingozzi F, Carlier P, Gjata B, Hogrel JY, Adjali O, Mavilio F, Voit T, Moullier P, Dickson G.

Nat Commun. 2017 Jul 25;8:16105. doi: 10.1038/ncomms16105.

10.

Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult.

Morris EC, Fox T, Chakraverty R, Tendeiro R, Snell K, Rivat C, Grace S, Gilmour K, Workman S, Buckland K, Butler K, Chee R, Salama AD, Ibrahim H, Hara H, Duret C, Mavilio F, Male F, Bushman FD, Galy A, Burns SO, Gaspar HB, Thrasher AJ.

Blood. 2017 Sep 14;130(11):1327-1335. doi: 10.1182/blood-2017-04-777136. Epub 2017 Jul 17.

PMID:
28716862
11.

Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System.

Lattanzi A, Duguez S, Moiani A, Izmiryan A, Barbon E, Martin S, Mamchaoui K, Mouly V, Bernardi F, Mavilio F, Bovolenta M.

Mol Ther Nucleic Acids. 2017 Jun 16;7:11-19. doi: 10.1016/j.omtn.2017.02.004. Epub 2017 Feb 10.

12.

Retroviral Scanning: Mapping MLV Integration Sites to Define Cell-specific Regulatory Regions.

Romano O, Cifola I, Poletti V, Severgnini M, Peano C, De Bellis G, Mavilio F, Miccio A.

J Vis Exp. 2017 May 28;(123). doi: 10.3791/55919.

PMID:
28605390
13.

Developing gene and cell therapies for rare diseases: an opportunity for synergy between academia and industry.

Mavilio F.

Gene Ther. 2017 Sep;24(9):590-592. doi: 10.1038/gt.2017.36. Epub 2017 May 25. Review.

PMID:
28485723
14.

Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs.

Mack DL, Poulard K, Goddard MA, Latournerie V, Snyder JM, Grange RW, Elverman MR, Denard J, Veron P, Buscara L, Le Bec C, Hogrel JY, Brezovec AG, Meng H, Yang L, Liu F, O'Callaghan M, Gopal N, Kelly VE, Smith BK, Strande JL, Mavilio F, Beggs AH, Mingozzi F, Lawlor MW, Buj-Bello A, Childers MK.

Mol Ther. 2017 Apr 5;25(4):839-854. doi: 10.1016/j.ymthe.2017.02.004. Epub 2017 Feb 22.

15.

Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy.

Armbruster N, Lattanzi A, Jeavons M, Van Wittenberghe L, Gjata B, Marais T, Martin S, Vignaud A, Voit T, Mavilio F, Barkats M, Buj-Bello A.

Mol Ther Methods Clin Dev. 2016 Sep 14;3:16060. doi: 10.1038/mtm.2016.60. eCollection 2016.

16.

Transcriptional, epigenetic and retroviral signatures identify regulatory regions involved in hematopoietic lineage commitment.

Romano O, Peano C, Tagliazucchi GM, Petiti L, Poletti V, Cocchiarella F, Rizzi E, Severgnini M, Cavazza A, Rossi C, Pagliaro P, Ambrosi A, Ferrari G, Bicciato S, De Bellis G, Mavilio F, Miccio A.

Sci Rep. 2016 Apr 20;6:24724. doi: 10.1038/srep24724.

17.

Dynamic Transcriptional and Epigenetic Regulation of Human Epidermal Keratinocyte Differentiation.

Cavazza A, Miccio A, Romano O, Petiti L, Malagoli Tagliazucchi G, Peano C, Severgnini M, Rizzi E, De Bellis G, Bicciato S, Mavilio F.

Stem Cell Reports. 2016 Apr 12;6(4):618-632. doi: 10.1016/j.stemcr.2016.03.003. Epub 2016 Mar 31.

18.

Genome-Wide Definition of Promoter and Enhancer Usage during Neural Induction of Human Embryonic Stem Cells.

Poletti V, Delli Carri A, Malagoli Tagliazucchi G, Faedo A, Petiti L, Mazza EM, Peano C, De Bellis G, Bicciato S, Miccio A, Cattaneo E, Mavilio F.

PLoS One. 2015 May 15;10(5):e0126590. doi: 10.1371/journal.pone.0126590. eCollection 2015.

19.

Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome.

Hacein-Bey Abina S, Gaspar HB, Blondeau J, Caccavelli L, Charrier S, Buckland K, Picard C, Six E, Himoudi N, Gilmour K, McNicol AM, Hara H, Xu-Bayford J, Rivat C, Touzot F, Mavilio F, Lim A, Treluyer JM, Héritier S, Lefrère F, Magalon J, Pengue-Koyi I, Honnet G, Blanche S, Sherman EA, Male F, Berry C, Malani N, Bushman FD, Fischer A, Thrasher AJ, Galy A, Cavazzana M.

JAMA. 2015 Apr 21;313(15):1550-63. doi: 10.1001/jama.2015.3253.

20.

Nuclear architecture dictates HIV-1 integration site selection.

Marini B, Kertesz-Farkas A, Ali H, Lucic B, Lisek K, Manganaro L, Pongor S, Luzzati R, Recchia A, Mavilio F, Giacca M, Lusic M.

Nature. 2015 May 14;521(7551):227-31. doi: 10.1038/nature14226. Epub 2015 Mar 2.

PMID:
25731161
21.

A single epidermal stem cell strategy for safe ex vivo gene therapy.

Droz-Georget Lathion S, Rochat A, Knott G, Recchia A, Martinet D, Benmohammed S, Grasset N, Zaffalon A, Besuchet Schmutz N, Savioz-Dayer E, Beckmann JS, Rougemont J, Mavilio F, Barrandon Y.

EMBO Mol Med. 2015 Apr;7(4):380-93. doi: 10.15252/emmm.201404353.

22.

Perspectives on best practices for gene therapy programs.

Cheever TR, Berkley D, Braun S, Brown RH, Byrne BJ, Chamberlain JS, Cwik V, Duan D, Federoff HJ, High KA, Kaspar BK, Klinger KW, Larkindale J, Lincecum J, Mavilio F, McDonald CL, McLaughlin J, Weiss McLeod B, Mendell JR, Nuckolls G, Stedman HH, Tagle DA, Vandenberghe LH, Wang H, Wernett PJ, Wilson JM, Porter JD, Gubitz AK.

Hum Gene Ther. 2015 Mar;26(3):127-33. doi: 10.1089/hum.2014.147. Epub 2015 Mar 3.

23.

Genomic analysis of Sleeping Beauty transposon integration in human somatic cells.

Turchiano G, Latella MC, Gogol-Döring A, Cattoglio C, Mavilio F, Izsvák Z, Ivics Z, Recchia A.

PLoS One. 2014 Nov 12;9(11):e112712. doi: 10.1371/journal.pone.0112712. eCollection 2014.

24.

Genome-wide analysis of alpharetroviral integration in human hematopoietic stem/progenitor cells.

Moiani A, Suerth JD, Gandolfi F, Rizzi E, Severgnini M, De Bellis G, Schambach A, Mavilio F.

Genes (Basel). 2014 May 16;5(2):415-29. doi: 10.3390/genes5020415.

25.

Repairing without cutting: a safer alternative to gene correction?

Mavilio F.

Mol Ther. 2014 Apr;22(4):690-1. doi: 10.1038/mt.2014.40. No abstract available.

26.

Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy.

Childers MK, Joubert R, Poulard K, Moal C, Grange RW, Doering JA, Lawlor MW, Rider BE, Jamet T, Danièle N, Martin S, Rivière C, Soker T, Hammer C, Van Wittenberghe L, Lockard M, Guan X, Goddard M, Mitchell E, Barber J, Williams JK, Mack DL, Furth ME, Vignaud A, Masurier C, Mavilio F, Moullier P, Beggs AH, Buj-Bello A.

Sci Transl Med. 2014 Jan 22;6(220):220ra10. doi: 10.1126/scitranslmed.3007523.

27.

RD2-MolPack-Chim3, a packaging cell line for stable production of lentiviral vectors for anti-HIV gene therapy.

Stornaiuolo A, Piovani BM, Bossi S, Zucchelli E, Corna S, Salvatori F, Mavilio F, Bordignon C, Rizzardi GP, Bovolenta C.

Hum Gene Ther Methods. 2013 Aug;24(4):228-40. doi: 10.1089/hgtb.2012.190. Epub 2013 Aug 3.

28.

Targeted gene addition in human epithelial stem cells by zinc-finger nuclease-mediated homologous recombination.

Coluccio A, Miselli F, Lombardo A, Marconi A, Malagoli Tagliazucchi G, Gonçalves MA, Pincelli C, Maruggi G, Del Rio M, Naldini L, Larcher F, Mavilio F, Recchia A.

Mol Ther. 2013 Sep;21(9):1695-704. doi: 10.1038/mt.2013.143. Epub 2013 Jun 13.

29.

Self-inactivating MLV vectors have a reduced genotoxic profile in human epidermal keratinocytes.

Cavazza A, Cocchiarella F, Bartholomae C, Schmidt M, Pincelli C, Larcher F, Mavilio F.

Gene Ther. 2013 Sep;20(9):949-57. doi: 10.1038/gt.2013.18. Epub 2013 Apr 25.

PMID:
23615186
30.

Genotoxic signature in cord blood cells of newborns exposed in utero to a Zidovudine-based antiretroviral combination.

André-Schmutz I, Dal-Cortivo L, Six E, Kaltenbach S, Cocchiarella F, Le Chenadec J, Cagnard N, Cordier AG, Benachi A, Mandelbrot L, Azria E, Bouallag N, Luce S, Ternaux B, Reimann C, Revy P, Radford-Weiss I, Leschi C, Recchia A, Mavilio F, Cavazzana M, Blanche S.

J Infect Dis. 2013 Jul 15;208(2):235-43. doi: 10.1093/infdis/jit149. Epub 2013 Apr 4.

PMID:
23559464
31.

Nup153 and Nup98 bind the HIV-1 core and contribute to the early steps of HIV-1 replication.

Di Nunzio F, Fricke T, Miccio A, Valle-Casuso JC, Perez P, Souque P, Rizzi E, Severgnini M, Mavilio F, Charneau P, Diaz-Griffero F.

Virology. 2013 May 25;440(1):8-18. doi: 10.1016/j.virol.2013.02.008. Epub 2013 Mar 21.

32.

Deletion of the LTR enhancer/promoter has no impact on the integration profile of MLV vectors in human hematopoietic progenitors.

Moiani A, Miccio A, Rizzi E, Severgnini M, Pellin D, Suerth JD, Baum C, De Bellis G, Mavilio F.

PLoS One. 2013;8(1):e55721. doi: 10.1371/journal.pone.0055721. Epub 2013 Jan 31.

33.

Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases.

Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, Boycott K, Braun S, Breakefield XO, Bueren JA, Buschmann M, Byrne BJ, Calos M, Cathomen T, Chamberlain J, Chuah M, Cornetta K, Davies KE, Dickson JG, Duchateau P, Flotte TR, Gaudet D, Gersbach CA, Gilbert R, Glorioso J, Herzog RW, High KA, Huang W, Huard J, Joung JK, Liu D, Liu D, Lochmüller H, Lustig L, Martens J, Massie B, Mavilio F, Mendell JR, Nathwani A, Ponder K, Porteus M, Puymirat J, Samulski J, Takeda S, Thrasher A, VandenDriessche T, Wei Y, Wilson JM, Wilton SD, Wolfe JH, Gao G.

Mol Ther. 2013 Feb;21(2):266-8. doi: 10.1038/mt.2013.4. No abstract available.

34.

Mechanisms of retroviral integration and mutagenesis.

Cavazza A, Moiani A, Mavilio F.

Hum Gene Ther. 2013 Feb;24(2):119-31. doi: 10.1089/hum.2012.203. Review.

PMID:
23330935
35.

IL-7 and IL-15 instruct the generation of human memory stem T cells from naive precursors.

Cieri N, Camisa B, Cocchiarella F, Forcato M, Oliveira G, Provasi E, Bondanza A, Bordignon C, Peccatori J, Ciceri F, Lupo-Stanghellini MT, Mavilio F, Mondino A, Bicciato S, Recchia A, Bonini C.

Blood. 2013 Jan 24;121(4):573-84. doi: 10.1182/blood-2012-05-431718. Epub 2012 Nov 15.

36.

Gene therapies need new development models.

Mavilio F.

Nature. 2012 Oct 4;490(7418):7. doi: 10.1038/490007a. No abstract available.

PMID:
23038429
37.

Lentiviral vector integration in the human genome induces alternative splicing and generates aberrant transcripts.

Moiani A, Paleari Y, Sartori D, Mezzadra R, Miccio A, Cattoglio C, Cocchiarella F, Lidonnici MR, Ferrari G, Mavilio F.

J Clin Invest. 2012 May;122(5):1653-66. doi: 10.1172/JCI61852. Epub 2012 Apr 23.

38.

Alternative splicing caused by lentiviral integration in the human genome.

Moiani A, Mavilio F.

Methods Enzymol. 2012;507:155-69. doi: 10.1016/B978-0-12-386509-0.00008-9.

PMID:
22365773
39.

Gene therapy of skin adhesion disorders (mini review).

Cavazza A, Mavilio F.

Curr Pharm Biotechnol. 2012 Aug;13(10):1868-76. Review.

PMID:
22250710
40.

The GATA1-HS2 enhancer allows persistent and position-independent expression of a β-globin transgene.

Miccio A, Poletti V, Tiboni F, Rossi C, Antonelli A, Mavilio F, Ferrari G.

PLoS One. 2011;6(12):e27955. doi: 10.1371/journal.pone.0027955. Epub 2011 Dec 2.

41.

Estimated comparative integration hotspots identify different behaviors of retroviral gene transfer vectors.

Ambrosi A, Glad IK, Pellin D, Cattoglio C, Mavilio F, Di Serio C, Frigessi A.

PLoS Comput Biol. 2011 Dec;7(12):e1002292. doi: 10.1371/journal.pcbi.1002292. Epub 2011 Dec 1.

42.

Preclinical corrective gene transfer in xeroderma pigmentosum human skin stem cells.

Warrick E, Garcia M, Chagnoleau C, Chevallier O, Bergoglio V, Sartori D, Mavilio F, Angulo JF, Avril MF, Sarasin A, Larcher F, Del Rio M, Bernerd F, Magnaldo T.

Mol Ther. 2012 Apr;20(4):798-807. doi: 10.1038/mt.2011.233. Epub 2011 Nov 8.

43.

Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy.

Deichmann A, Brugman MH, Bartholomae CC, Schwarzwaelder K, Verstegen MM, Howe SJ, Arens A, Ott MG, Hoelzer D, Seger R, Grez M, Hacein-Bey-Abina S, Cavazzana-Calvo M, Fischer A, Paruzynski A, Gabriel R, Glimm H, Abel U, Cattoglio C, Mavilio F, Cassani B, Aiuti A, Dunbar CE, Baum C, Gaspar HB, Thrasher AJ, von Kalle C, Schmidt M, Wagemaker G.

Mol Ther. 2011 Nov;19(11):2031-9. doi: 10.1038/mt.2011.178. Epub 2011 Aug 23.

44.

Site-specific integration by the adeno-associated virus rep protein.

Recchia A, Mavilio F.

Curr Gene Ther. 2011 Oct;11(5):399-405. Review.

PMID:
21827397
45.

Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning.

Huston MW, van Til NP, Visser TP, Arshad S, Brugman MH, Cattoglio C, Nowrouzi A, Li Y, Schambach A, Schmidt M, Baum C, von Kalle C, Mavilio F, Zhang F, Blundell MP, Thrasher AJ, Verstegen MM, Wagemaker G.

Mol Ther. 2011 Oct;19(10):1867-77. doi: 10.1038/mt.2011.127. Epub 2011 Jul 12.

46.

Risk assessment in skin gene therapy: viral-cellular fusion transcripts generated by proviral transcriptional read-through in keratinocytes transduced with self-inactivating lentiviral vectors.

Almarza D, Bussadori G, Navarro M, Mavilio F, Larcher F, Murillas R.

Gene Ther. 2011 Jul;18(7):674-81. doi: 10.1038/gt.2011.12. Epub 2011 Mar 3.

PMID:
21368897
47.

High-definition mapping of retroviral integration sites defines the fate of allogeneic T cells after donor lymphocyte infusion.

Cattoglio C, Maruggi G, Bartholomae C, Malani N, Pellin D, Cocchiarella F, Magnani Z, Ciceri F, Ambrosi A, von Kalle C, Bushman FD, Bonini C, Schmidt M, Mavilio F, Recchia A.

PLoS One. 2010 Dec 22;5(12):e15688. doi: 10.1371/journal.pone.0015688.

48.

High-definition mapping of retroviral integration sites identifies active regulatory elements in human multipotent hematopoietic progenitors.

Cattoglio C, Pellin D, Rizzi E, Maruggi G, Corti G, Miselli F, Sartori D, Guffanti A, Di Serio C, Ambrosi A, De Bellis G, Mavilio F.

Blood. 2010 Dec 16;116(25):5507-17. doi: 10.1182/blood-2010-05-283523. Epub 2010 Sep 23.

49.

Correction of beta-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patients.

Roselli EA, Mezzadra R, Frittoli MC, Maruggi G, Biral E, Mavilio F, Mastropietro F, Amato A, Tonon G, Refaldi C, Cappellini MD, Andreani M, Lucarelli G, Roncarolo MG, Marktel S, Ferrari G.

EMBO Mol Med. 2010 Aug;2(8):315-28. doi: 10.1002/emmm.201000083.

50.

Gene therapy: back on track?

Mavilio F.

EMBO Rep. 2010 Feb;11(2):75. doi: 10.1038/embor.2009.284. No abstract available.

Supplemental Content

Loading ...
Support Center