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Items: 1 to 50 of 106

1.

Deep Dermal Injection As a Model of Candida albicans Skin Infection for Histological Analyses.

Santus W, Mingozzi F, Vai M, Granucci F, Zanoni I.

J Vis Exp. 2018 Jun 13;(136). doi: 10.3791/57574.

PMID:
29985352
2.

Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction.

Fitzpatrick Z, Leborgne C, Barbon E, Masat E, Ronzitti G, van Wittenberghe L, Vignaud A, Collaud F, Charles S, Simon Sola M, Jouen F, Boyer O, Mingozzi F.

Mol Ther Methods Clin Dev. 2018 Feb 13;9:119-129. doi: 10.1016/j.omtm.2018.02.003. eCollection 2018 Jun 15.

3.

Exon-specific U1 snRNAs improve ELP1 exon 20 definition and rescue ELP1 protein expression in a familial dysautonomia mouse model.

Donadon I, Pinotti M, Rajkowska K, Pianigiani G, Barbon E, Morini E, Motaln H, Rogelj B, Mingozzi F, Slaugenhaupt SA, Pagani F.

Hum Mol Genet. 2018 Jul 15;27(14):2466-2476. doi: 10.1093/hmg/ddy151.

4.

Author Correction: μMAPPS: a novel phasor approach to second harmonic analysis for in vitro-in vivo investigation of collagen microstructure.

Radaelli F, D'Alfonso L, Collini M, Mingozzi F, Marongiu L, Granucci F, Zanoni I, Chirico G, Sironi L.

Sci Rep. 2018 Apr 17;8(1):6314. doi: 10.1038/s41598-018-24800-6.

5.

Prevalence and long-term monitoring of humoral immunity against adeno-associated virus in Duchenne Muscular Dystrophy patients.

Leborgne C, Latournerie V, Boutin S, Desgue D, Quéré A, Pignot E, Collaud F, Charles S, Simon Sola M, Masat E, Jouen F, Boyer O, Masurier C, Mingozzi F, Veron P.

Cell Immunol. 2018 Mar 16. pii: S0008-8749(18)30114-X. doi: 10.1016/j.cellimm.2018.03.004. [Epub ahead of print]

PMID:
29571923
6.

Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression.

Vidal P, Pagliarani S, Colella P, Costa Verdera H, Jauze L, Gjorgjieva M, Puzzo F, Marmier S, Collaud F, Simon Sola M, Charles S, Lucchiari S, van Wittenberghe L, Vignaud A, Gjata B, Richard I, Laforet P, Malfatti E, Mithieux G, Rajas F, Comi GP, Ronzitti G, Mingozzi F.

Mol Ther. 2018 Mar 7;26(3):890-901. doi: 10.1016/j.ymthe.2017.12.019. Epub 2017 Dec 28.

PMID:
29396266
7.

Emerging Issues in AAV-Mediated In Vivo Gene Therapy.

Colella P, Ronzitti G, Mingozzi F.

Mol Ther Methods Clin Dev. 2017 Dec 1;8:87-104. doi: 10.1016/j.omtm.2017.11.007. eCollection 2018 Mar 16. Review.

8.

Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors.

Meliani A, Boisgerault F, Fitzpatrick Z, Marmier S, Leborgne C, Collaud F, Simon Sola M, Charles S, Ronzitti G, Vignaud A, van Wittenberghe L, Marolleau B, Jouen F, Tan S, Boyer O, Christophe O, Brisson AR, Maguire CA, Mingozzi F.

Blood Adv. 2017 Oct 16;1(23):2019-2031. doi: 10.1182/bloodadvances.2017010181. eCollection 2017 Oct 24.

9.

Combination Therapy Is the New Gene Therapy?

Ronzitti G, Mingozzi F.

Mol Ther. 2018 Jan 3;26(1):12-14. doi: 10.1016/j.ymthe.2017.12.008. Epub 2017 Dec 20. No abstract available.

PMID:
29273499
10.

μMAPPS: a novel phasor approach to second harmonic analysis for in vitro-in vivo investigation of collagen microstructure.

Radaelli F, D'Alfonso L, Collini M, Mingozzi F, Marongiu L, Granucci F, Zanoni I, Chirico G, Sironi L.

Sci Rep. 2017 Dec 12;7(1):17468. doi: 10.1038/s41598-017-17726-y. Erratum in: Sci Rep. 2018 Apr 17;8(1):6314.

11.

Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.

Puzzo F, Colella P, Biferi MG, Bali D, Paulk NK, Vidal P, Collaud F, Simon-Sola M, Charles S, Hardet R, Leborgne C, Meliani A, Cohen-Tannoudji M, Astord S, Gjata B, Sellier P, van Wittenberghe L, Vignaud A, Boisgerault F, Barkats M, Laforet P, Kay MA, Koeberl DD, Ronzitti G, Mingozzi F.

Sci Transl Med. 2017 Nov 29;9(418). pii: eaam6375. doi: 10.1126/scitranslmed.aam6375.

12.

Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity.

Paulk NK, Pekrun K, Zhu E, Nygaard S, Li B, Xu J, Chu K, Leborgne C, Dane AP, Haft A, Zhang Y, Zhang F, Morton C, Valentine MB, Davidoff AM, Nathwani AC, Mingozzi F, Grompe M, Alexander IE, Lisowski L, Kay MA.

Mol Ther. 2018 Jan 3;26(1):289-303. doi: 10.1016/j.ymthe.2017.09.021. Epub 2017 Sep 25.

PMID:
29055620
13.

Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape.

Mingozzi F, High KA.

Annu Rev Virol. 2017 Sep 29;4(1):511-534. doi: 10.1146/annurev-virology-101416-041936. Review.

PMID:
28961410
14.

Skin infections are eliminated by cooperation of the fibrinolytic and innate immune systems.

Santus W, Barresi S, Mingozzi F, Broggi A, Orlandi I, Stamerra G, Vai M, Martorana AM, Polissi A, Köhler JR, Liu N, Zanoni I, Granucci F.

Sci Immunol. 2017 Sep 22;2(15). pii: eaan2725. doi: 10.1126/sciimmunol.aan2725.

PMID:
28939652
15.

Unraveling the Complex Story of Immune Responses to AAV Vectors Trial After Trial.

Vandamme C, Adjali O, Mingozzi F.

Hum Gene Ther. 2017 Nov;28(11):1061-1074. doi: 10.1089/hum.2017.150. Review.

16.

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.

17.

Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction.

Han SO, Ronzitti G, Arnson B, Leborgne C, Li S, Mingozzi F, Koeberl D.

Mol Ther Methods Clin Dev. 2017 Jan 11;4:126-136. doi: 10.1016/j.omtm.2016.12.010. eCollection 2017 Mar 17.

18.

Autophagy determines efficiency of liver-directed gene therapy with adeno-associated viral vectors.

Hösel M, Huber A, Bohlen S, Lucifora J, Ronzitti G, Puzzo F, Boisgerault F, Hacker UT, Kwanten WJ, Klöting N, Blüher M, Gluschko A, Schramm M, Utermöhlen O, Bloch W, Mingozzi F, Krut O, Büning H.

Hepatology. 2017 Jul;66(1):252-265. doi: 10.1002/hep.29176. Epub 2017 May 29.

19.

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.

20.

Oral Tolerance: Another Reason to Eat Your Veggies!

Hardet R, Mingozzi F.

Mol Ther. 2017 Feb 1;25(2):311-313. doi: 10.1016/j.ymthe.2017.01.001. Epub 2017 Jan 18. No abstract available.

21.

Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics.

Barbon E, Ferrarese M, van Wittenberghe L, Sanatine P, Ronzitti G, Collaud F, Colella P, Pinotti M, Mingozzi F.

Mol Ther Nucleic Acids. 2016 Nov 29;5(11):e392. doi: 10.1038/mtna.2016.97.

22.

Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients.

Masat E, Laforêt P, De Antonio M, Corre G, Perniconi B, Taouagh N, Mariampillai K, Amelin D, Mauhin W, Hogrel JY, Caillaud C, Ronzitti G, Puzzo F, Kuranda K, Colella P, Mallone R, Benveniste O, Mingozzi F; French Pompe Registry Study Group.

Sci Rep. 2016 Nov 4;6:36182. doi: 10.1038/srep36182.

23.

Exosome-associated AAV vector as a robust and convenient neuroscience tool.

Hudry E, Martin C, Gandhi S, György B, Scheffer DI, Mu D, Merkel SF, Mingozzi F, Fitzpatrick Z, Dimant H, Masek M, Ragan T, Tan S, Brisson AR, Ramirez SH, Hyman BT, Maguire CA.

Gene Ther. 2016 Nov;23(11):819. doi: 10.1038/gt.2016.65. No abstract available.

PMID:
27808124
24.

A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome.

Ronzitti G, Bortolussi G, van Dijk R, Collaud F, Charles S, Leborgne C, Vidal P, Martin S, Gjata B, Sola MS, van Wittenberghe L, Vignaud A, Veron P, Bosma PJ, Muro AF, Mingozzi F.

Mol Ther Methods Clin Dev. 2016 Jul 20;3:16049. eCollection 2016.

25.

Induction of Hematopoietic Microchimerism by Gene-Modified BMT Elicits Antigen-Specific B and T Cell Unresponsiveness toward Gene Therapy Products.

Martinet J, Bourdenet G, Meliani A, Jean L, Adriouch S, Cohen JL, Mingozzi F, Boyer O.

Front Immunol. 2016 Sep 15;7:360. eCollection 2016.

26.

Prolonged contact with dendritic cells turns lymph node-resident NK cells into anti-tumor effectors.

Mingozzi F, Spreafico R, Gorletta T, Cigni C, Di Gioia M, Caccia M, Sironi L, Collini M, Soncini M, Rusconi M, von Andrian UH, Chirico G, Zanoni I, Granucci F.

EMBO Mol Med. 2016 Sep 1;8(9):1039-51. doi: 10.15252/emmm.201506164. Print 2016 Sep.

27.

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.

Bennett J, Wellman J, Marshall KA, McCague S, Ashtari M, DiStefano-Pappas J, Elci OU, Chung DC, Sun J, Wright JF, Cross DR, Aravand P, Cyckowski LL, Bennicelli JL, Mingozzi F, Auricchio A, Pierce EA, Ruggiero J, Leroy BP, Simonelli F, High KA, Maguire AM.

Lancet. 2016 Aug 13;388(10045):661-72. doi: 10.1016/S0140-6736(16)30371-3. Epub 2016 Jun 30.

28.

Exosome-associated AAV vector as a robust and convenient neuroscience tool.

Hudry E, Martin C, Gandhi S, György B, Scheffer DI, Mu D, Merkel SF, Mingozzi F, Fitzpatrick Z, Dimant H, Masek M, Ragan T, Tan S, Brisson AR, Ramirez SH, Hyman BT, Maguire CA.

Gene Ther. 2016 Apr;23(4):380-92. doi: 10.1038/gt.2016.11. Epub 2016 Feb 2. Erratum in: Gene Ther. 2016 Nov;23 (11):819.

29.

AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes.

Hui DJ, Edmonson SC, Podsakoff GM, Pien GC, Ivanciu L, Camire RM, Ertl H, Mingozzi F, High KA, Basner-Tschakarjan E.

Mol Ther Methods Clin Dev. 2015 Sep 30;2:15029. doi: 10.1038/mtm.2015.29. eCollection 2015.

30.
31.

Preclinical Potency and Biodistribution Studies of an AAV 5 Vector Expressing Human Interferon-β (ART-I02) for Local Treatment of Patients with Rheumatoid Arthritis.

Aalbers CJ, Bevaart L, Loiler S, de Cortie K, Wright JF, Mingozzi F, Tak PP, Vervoordeldonk MJ.

PLoS One. 2015 Jun 24;10(6):e0130612. doi: 10.1371/journal.pone.0130612. eCollection 2015.

32.

Adeno-Associated Viral Vectors at the Frontier between Tolerance and Immunity.

Mingozzi F, Büning H.

Front Immunol. 2015 Mar 17;6:120. doi: 10.3389/fimmu.2015.00120. eCollection 2015. No abstract available.

33.

Determination of anti-adeno-associated virus vector neutralizing antibody titer with an in vitro reporter system.

Meliani A, Leborgne C, Triffault S, Jeanson-Leh L, Veron P, Mingozzi F.

Hum Gene Ther Methods. 2015 Apr;26(2):45-53. doi: 10.1089/hgtb.2015.037.

34.

Innate and Adaptive Immune Response in Fabry Disease.

Mauhin W, Lidove O, Masat E, Mingozzi F, Mariampillai K, Ziza JM, Benveniste O.

JIMD Rep. 2015;22:1-10. doi: 10.1007/8904_2014_371. Epub 2015 Feb 18.

35.

Adeno-associated virus vector-based gene therapy for monogenetic metabolic diseases of the liver.

Junge N, Mingozzi F, Ott M, Baumann U.

J Pediatr Gastroenterol Nutr. 2015 Apr;60(4):433-40. doi: 10.1097/MPG.0000000000000703. Review.

PMID:
25594875
36.

AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice.

Crudele JM, Finn JD, Siner JI, Martin NB, Niemeyer GP, Zhou S, Mingozzi F, Lothrop CD Jr, Arruda VR.

Blood. 2015 Mar 5;125(10):1553-61. doi: 10.1182/blood-2014-07-588194. Epub 2015 Jan 7.

37.

Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy.

Ribera A, Haurigot V, Garcia M, Marcó S, Motas S, Villacampa P, Maggioni L, León X, Molas M, Sánchez V, Muñoz S, Leborgne C, Moll X, Pumarola M, Mingozzi F, Ruberte J, Añor S, Bosch F.

Hum Mol Genet. 2015 Apr 1;24(7):2078-95. doi: 10.1093/hmg/ddu727. Epub 2014 Dec 18.

PMID:
25524704
38.

Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

Nathwani AC, Reiss UM, Tuddenham EG, Rosales C, Chowdary P, McIntosh J, Della Peruta M, Lheriteau E, Patel N, Raj D, Riddell A, Pie J, Rangarajan S, Bevan D, Recht M, Shen YM, Halka KG, Basner-Tschakarjan E, Mingozzi F, High KA, Allay J, Kay MA, Ng CY, Zhou J, Cancio M, Morton CL, Gray JT, Srivastava D, Nienhuis AW, Davidoff AM.

N Engl J Med. 2014 Nov 20;371(21):1994-2004. doi: 10.1056/NEJMoa1407309.

39.

Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade.

Han SO, Li S, Brooks ED, Masat E, Leborgne C, Banugaria S, Bird A, Mingozzi F, Waldmann H, Koeberl D.

Hum Gene Ther. 2015 Jan;26(1):26-35. doi: 10.1089/hum.2014.115.

40.

Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions.

Basner-Tschakarjan E, Mingozzi F.

Front Immunol. 2014 Jul 23;5:350. doi: 10.3389/fimmu.2014.00350. eCollection 2014. Review.

41.

Liver-specific transcriptional modules identified by genome-wide in silico analysis enable efficient gene therapy in mice and non-human primates.

Chuah MK, Petrus I, De Bleser P, Le Guiner C, Gernoux G, Adjali O, Nair N, Willems J, Evens H, Rincon MY, Matrai J, Di Matteo M, Samara-Kuko E, Yan B, Acosta-Sanchez A, Meliani A, Cherel G, Blouin V, Christophe O, Moullier P, Mingozzi F, VandenDriessche T.

Mol Ther. 2014 Sep;22(9):1605-13. doi: 10.1038/mt.2014.114. Epub 2014 Jun 23.

42.

Th1 response and systemic treg deficiency in inclusion body myositis.

Allenbach Y, Chaara W, Rosenzwajg M, Six A, Prevel N, Mingozzi F, Wanschitz J, Musset L, Charuel JL, Eymard B, Salomon B, Duyckaerts C, Maisonobe T, Dubourg O, Herson S, Klatzmann D, Benveniste O.

PLoS One. 2014 Mar 4;9(3):e88788. doi: 10.1371/journal.pone.0088788. eCollection 2014.

43.

Tregitope peptides: the active pharmaceutical ingredient of IVIG?

De Groot AS, Cousens L, Mingozzi F, Martin W.

Clin Dev Immunol. 2013;2013:493138. doi: 10.1155/2013/493138. Epub 2013 Dec 25. Review.

44.

Overcoming preexisting humoral immunity to AAV using capsid decoys.

Mingozzi F, Anguela XM, Pavani G, Chen Y, Davidson RJ, Hui DJ, Yazicioglu M, Elkouby L, Hinderer CJ, Faella A, Howard C, Tai A, Podsakoff GM, Zhou S, Basner-Tschakarjan E, Wright JF, High KA.

Sci Transl Med. 2013 Jul 17;5(194):194ra92. doi: 10.1126/scitranslmed.3005795.

45.

Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy.

Haurigot V, Marcó S, Ribera A, Garcia M, Ruzo A, Villacampa P, Ayuso E, Añor S, Andaluz A, Pineda M, García-Fructuoso G, Molas M, Maggioni L, Muñoz S, Motas S, Ruberte J, Mingozzi F, Pumarola M, Bosch F.

J Clin Invest. 2013 Jul 1. pii: 66778. doi: 10.1172/JCI66778. [Epub ahead of print]

46.

Modulation of CD8+ T cell responses to AAV vectors with IgG-derived MHC class II epitopes.

Hui DJ, Basner-Tschakarjan E, Chen Y, Davidson RJ, Buchlis G, Yazicioglu M, Pien GC, Finn JD, Haurigot V, Tai A, Scott DW, Cousens LP, Zhou S, De Groot AS, Mingozzi F.

Mol Ther. 2013 Sep;21(9):1727-37. doi: 10.1038/mt.2013.166. Epub 2013 Jul 16.

47.

IL12-mediated liver inflammation reduces the formation of AAV transcriptionally active forms but has no effect over preexisting AAV transgene expression.

Gil-Fariña I, Di Scala M, Vanrell L, Olagüe C, Vales A, High KA, Prieto J, Mingozzi F, Gonzalez-Aseguinolaza G.

PLoS One. 2013 Jul 2;8(7):e67748. doi: 10.1371/journal.pone.0067748. Print 2013.

48.

Humoral immunity to AAV vectors in gene therapy: challenges and potential solutions.

Masat E, Pavani G, Mingozzi F.

Discov Med. 2013 Jun;15(85):379-89. Review.

49.

Immune responses to AAV vectors: overcoming barriers to successful gene therapy.

Mingozzi F, High KA.

Blood. 2013 Jul 4;122(1):23-36. doi: 10.1182/blood-2013-01-306647. Epub 2013 Apr 17. Review.

50.

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant.

McIntosh J, Lenting PJ, Rosales C, Lee D, Rabbanian S, Raj D, Patel N, Tuddenham EG, Christophe OD, McVey JH, Waddington S, Nienhuis AW, Gray JT, Fagone P, Mingozzi F, Zhou SZ, High KA, Cancio M, Ng CY, Zhou J, Morton CL, Davidoff AM, Nathwani AC.

Blood. 2013 Apr 25;121(17):3335-44. doi: 10.1182/blood-2012-10-462200. Epub 2013 Feb 20.

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