2017

[13] J. Marsolier, P. Laforet, E. Pegoraro, J. Vissing, I. Richard, C. Barnerias, R. Y. Carlier, J. Diaz-Manera, A. Fayssoil, A. Galy, E. Gazzerro, D. Gorecki, M. Guglieri, J. Y. Hogrel, D. Israeli, F. Leturcq, H. Moussu, H. Prigent, D. Sandona, B. Schoser, C. Semplicini, B. Talim, G. Tasca, A. Urtizberea, and B. Udd. 1st International Workshop on Clinical trial readiness for sarcoglycanopathies 15-16 November 2016, Evry, France. Neuromuscul. Disord., Mar 2017. [DOI:10.1016/j.nmd.2017.02.011] [PubMed:28521973].
[12] A. Vihola, H. Luque, M. Savarese, S. Penttila, M. Lindfors, F. Leturcq, B. Eymard, G. Tasca, B. Brais, T. Conte, K. Charton, I. Richard, and B. Udd. Diagnostic anoctamin-5 protein defect in patients with ANO5-mutated muscular dystrophy. Neuropathol. Appl. Neurobiol., May 2017. [DOI:10.1111/nan.12410] [PubMed:28489263].
[11] F. Mavilio. Developing gene and cell therapies for rare diseases: an opportunity for synergy between academia and industry. Gene Ther., May 2017. [DOI:10.1038/gt.2017.36] [PubMed:28485723].
[10] C. Piovan, V. Marin, C. Scavullo, S. Corna, E. Giuliani, S. Bossi, A. Galy, D. Fenard, C. Bordignon, G. P. Rizzardi, and C. Bovolenta. Vectofusin-1 Promotes RD114-TR-Pseudotyped Lentiviral Vector Transduction of Human HSPCs and T Lymphocytes. Mol Ther Methods Clin Dev, 5:22-30, Jun 2017. [PubMed Central:PMC5415310] [DOI:10.1016/j.omtm.2017.02.003] [PubMed:28480301].
[9] M. Elverman, M. A. Goddard, D. Mack, J. M. Snyder, M. W. Lawlor, H. Meng, A. H. Beggs, A. Buj-Bello, K. Poulard, A. P. Marsh, R. W. Grange, V. E. Kelly, and M. K. Childers. Long-term effects of systemic gene therapy in a canine model of myotubular myopathy. Muscle Nerve, Mar 2017. [DOI:10.1002/mus.25658] [PubMed:28370029].
[8] S. O. Han, G. Ronzitti, B. Arnson, C. Leborgne, S. Li, F. Mingozzi, and D. Koeberl. Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction. Mol Ther Methods Clin Dev, 4:126-136, Mar 2017. [PubMed Central:PMC5363303] [DOI:10.1016/j.omtm.2016.12.010] [PubMed:28344998].
[7] E. Gicquel, N. Maizonnier, S. J. Foltz, W. J. Martin, N. Bourg, F. Svinartchouk, K. Charton, A. M. Beedle, and I. Richard. AAV-mediated transfer of FKRP shows therapeutic efficacy in a murine model but requires control of gene expression. Hum. Mol. Genet., 26(10):1952-1965, May 2017. [DOI:10.1093/hmg/ddx066] [PubMed:28334834].
[6] N. Nagy, R. J. Nonneman, T. Llanga, C. F. Dial, N. V. Riddick, T. Hampton, S. S. Moy, K. K. Lehtimaki, T. Ahtoniemi, J. Puolivali, H. Windish, D. Albrecht, I. Richard, and M. L. Hirsch. Hip region muscular dystrophy and emergence of motor deficits in dysferlin-deficient Bla/J mice. Physiol Rep, 5(6), Mar 2017. [PubMed Central:PMC5371557] [DOI:10.14814/phy2.13173] [PubMed:28320887].
[5] M. Hosel, A. Huber, S. Bohlen, J. Lucifora, G. Ronzitti, F. Puzzo, F. Boisgerault, U. T. Hacker, W. J. Kwanten, N. Kloting, M. Bluher, A. Gluschko, M. Schramm, O. Utermohlen, W. Bloch, F. Mingozzi, O. Krut, and H. Buning. Autophagy determines efficiency of liver-directed gene therapy with adeno-associated viral vectors. Hepatology, Mar 2017. [DOI:10.1002/hep.29176] [PubMed:28318036].
[4] J. Cosette, A. Moussy, A. Paldi, and D. Stockholm. Combination of imaging flow cytometry and time-lapse microscopy for the study of label-free morphology dynamics of hematopoietic cells. Cytometry A, 91(3):254-260, Mar 2017. [DOI:10.1002/cyto.a.23064] [PubMed:28248454].
[3] D. L. Mack, K. Poulard, M. A. Goddard, V. Latournerie, J. M. Snyder, R. W. Grange, M. R. Elverman, J. Denard, P. Veron, L. Buscara, C. Le Bec, J. Y. Hogrel, A. G. Brezovec, H. Meng, L. Yang, F. Liu, M. O'Callaghan, N. Gopal, V. E. Kelly, B. K. Smith, J. L. Strande, F. Mavilio, A. H. Beggs, F. Mingozzi, M. W. Lawlor, A. Buj-Bello, and M. K. Childers. Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs. Mol. Ther., 25(4):839-854, Apr 2017. [PubMed Central:PMC5383631] [DOI:10.1016/j.ymthe.2017.02.004] [PubMed:28237839].
[2] R. Hardet and F. Mingozzi. Oral Tolerance: Another Reason to Eat Your Veggies! Mol. Ther., 25(2):311-313, Feb 2017. [PubMed Central:PMC5368840] [DOI:10.1016/j.ymthe.2017.01.001] [PubMed:28109961].
[1] N. Holic, S. Frin, A. K. Seye, A. Galy, and D. Fenard. Improvement of De Novo Cholesterol Biosynthesis Efficiently Promotes the Production of Human Immunodeficiency Virus Type 1-Derived Lentiviral Vectors. Hum Gene Ther Methods, 28(2):67-77, Apr 2017. [DOI:10.1089/hgtb.2016.150] [PubMed:28042946].

2016

[33] N. Holic, S. Frin, A. K. Seye, A. Galy, and D. Fenard. Improvement of De Novo Cholesterol Biosynthesis Efficiently Promotes the Production of Human Immunodeficiency Virus Type 1-Derived Lentiviral Vectors. Hum Gene Ther Methods, Dec 2016. [DOI:10.1089/hgtb.2016.150] [PubMed:28042946].
[32] L. S. Vermeer, A. Marquette, M. Schoup, D. Fenard, A. Galy, and B. Bechinger. Simultaneous Analysis of Secondary Structure and Light Scattering from Circular Dichroism Titrations: Application to Vectofusin-1. Sci Rep, 6:39450, Dec 2016. [DOI:10.1038/srep39450] [PubMed:28004740].
[31] C. Kutchukian, M. Lo Scrudato, Y. Tourneur, K. Poulard, A. Vignaud, C. Berthier, B. Allard, M. W. Lawlor, A. Buj-Bello, and V. Jacquemond. Phosphatidylinositol 3-kinase inhibition restores Ca2+ release defects and prolongs survival in myotubularin-deficient mice. Proc. Natl. Acad. Sci. U.S.A., 113(50):14432-14437, Dec 2016. [PubMed Central:PMC5167204] [DOI:10.1073/pnas.1604099113] [PubMed:27911767].
[30] E. Barbon, M. Ferrarese, L. van Wittenberghe, P. Sanatine, G. Ronzitti, F. Collaud, P. Colella, M. Pinotti, and F. Mingozzi. Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics. Mol Ther Nucleic Acids, 5(11):e392, Nov 2016. [PubMed Central:PMC5155329] [DOI:10.1038/mtna.2016.97] [PubMed:27898092].
[29] N. El Shafey, M. Guesnon, F. Simon, E. Deprez, J. Cosette, D. Stockholm, D. Scherman, P. Bigey, and A. Kichler. Inhibition of the myostatin/Smad signaling pathway by short decorin-derived peptides. Exp. Cell Res., 341(2):187-195, Feb 2016. [DOI:10.1016/j.yexcr.2016.01.019] [PubMed:26844629].
[28] F. Nefzi, C. Lambert, A. Gautheret-Dejean, S. Fisson, Q. Khebizi, A. Khelif, H. Agut, and M. Aouni. The cytokines and cellular responses to human herpesvirus-6B in patients with B-acute lymphoblastic leukemia. Microbiol. Immunol., Nov 2016. [DOI:10.1111/1348-0421.12452] [PubMed:27862208].
[27] E. Hudry, C. Martin, S. Gandhi, B. Gyorgy, D. I. Scheffer, D. Mu, S. F. Merkel, F. Mingozzi, Z. Fitzpatrick, H. Dimant, M. Masek, T. Ragan, S. Tan, A. R. Brisson, S. H. Ramirez, B. T. Hyman, and C. A. Maguire. Exosome-associated AAV vector as a robust and convenient neuroscience tool. Gene Ther., 23(11):819, Nov 2016. [DOI:10.1038/gt.2016.65] [PubMed:27808124].
[26] E. Masat, P. Laforet, M. De Antonio, G. Corre, B. Perniconi, N. Taouagh, K. Mariampillai, D. Amelin, W. Mauhin, J. Y. Hogrel, C. Caillaud, G. Ronzitti, F. Puzzo, K. Kuranda, P. Colella, R. Mallone, O. Benveniste, F. Mingozzi, G. Bassez, A. L. Bedat-Millet, A. Behin, B. Eymard, S. Leonard-Louis, T. Stojkovic, A. Canal, V. Decostre, F. Bouhour, F. Boyer, Y. Castaing, F. Chapon, P. Cintas, I. Durieu, A. Echaniz-Laguna, L. Feasson, A. Furby, D. Hamroun, X. Ferrer, G. Sole, R. Froissart, M. Piraud, D. Germain, K. Benistan, N. Guffon-Fouilhoux, H. Journel, P. Labauge, A. Lacour, A. Levy, A. Magot, Y. Pereon, M. C. Minot-Myhie, A. Nadaj-Pakleza, C. Nathier, D. Orlikowski, N. Pellegrini, P. Petiot, J. Praline, F. Lofaso, H. Prigent, A. Dutry, D. Renard, S. Sacconi, C. Desnuelle, E. Salort-Campana, J. Pouget, V. Tiffreau, D. Vincent, and F. Zagnoli. Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients. Sci Rep, 6:36182, Nov 2016. [PubMed Central:PMC5096052] [DOI:10.1038/srep36182] [PubMed:27812025].
[25] I. Dalichaouche, Y. Sifi, C. Roudaut, K. Sifi, A. Hamri, L. Rouabah, N. Abadi, and I. Richard. γ-Sarcoglycan and Dystrophin Mutation Spectrum in an Algerian Cohort. Muscle Nerve, Oct 2016. [DOI:10.1002/mus.25443] [PubMed:27759885].
[24] G. Ronzitti, G. Bortolussi, R. van Dijk, F. Collaud, S. Charles, C. Leborgne, P. Vidal, S. Martin, B. Gjata, M. S. Sola, L. van Wittenberghe, A. Vignaud, P. Veron, P. J. Bosma, A. F. Muro, and F. Mingozzi. A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome. Mol Ther Methods Clin Dev, 3:16049, 2016. [DOI:10.1038/mtm.2016.49] [PubMed:27722180].
[23] J. Martinet, G. Bourdenet, A. Meliani, L. Jean, S. Adriouch, J. L. Cohen, F. Mingozzi, and O. Boyer. Induction of Hematopoietic Microchimerism by Gene-Modified BMT Elicits Antigen-Specific B and T Cell Unresponsiveness toward Gene Therapy Products. Front Immunol, 7:360, 2016. [DOI:10.3389/fimmu.2016.00360] [PubMed:27695454].
[22] N. Armbruster, A. Lattanzi, M. Jeavons, L. Van Wittenberghe, B. Gjata, T. Marais, S. Martin, A. Vignaud, T. Voit, F. Mavilio, M. Barkats, and A. Buj-Bello. Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy. Mol Ther Methods Clin Dev, 3:16060, 2016. [PubMed Central:PMC5022869] [DOI:10.1038/mtm.2016.60] [PubMed:27652289].
[21] A. Galy. Like Angler Fish, CAARs Lure Their Prey. Mol. Ther., 24(8):1339-1341, Aug 2016. [DOI:10.1038/mt.2016.165] [PubMed:27578283].
[20] S. Cire, S. Da Rocha, M. Ferrand, M. K. Collins, and A. Galy. In vivo gene delivery to lymph node stromal cells leads to transgene-specific CD8+ T cell anergy in mice. Mol. Ther., Aug 2016. [DOI:10.1038/mt.2016.168] [PubMed:27562586].
[19] K. Charton, L. Suel, S. F. Henriques, J. P. Moussu, M. Bovolenta, M. Taillepierre, C. Becker, K. Lipson, and I. Richard. Exploiting The CRISPR/CAS9 System to Study Alternative Splicing In Vivo: Application to Titin. Hum. Mol. Genet., Aug 2016. [DOI:10.1093/hmg/ddw280] [PubMed:27554112].
[18] N. Sabha, J. R. Volpatti, H. Gonorazky, A. Reifler, A. E. Davidson, X. Li, N. M. Eltayeb, C. Dall'Armi, G. Di Paolo, S. V. Brooks, A. Buj-Bello, E. L. Feldman, and J. J. Dowling. PIK3C2B inhibition improves function and prolongs survival in myotubular myopathy animal models. J. Clin. Invest., Aug 2016. [DOI:10.1172/JCI86841] [PubMed:27548528].
[17] J. Cosette, R. Ben Abdelwahed, S. Donnou-Triffault, C. Sautes-Fridman, P. Flaud, and S. Fisson. Bioluminescence-Based Tumor Quantification Method for Monitoring Tumor Progression and Treatment Effects in Mouse Lymphoma Models. J Vis Exp, (113), 2016. [DOI:10.3791/53609] [PubMed:27501019].
[16] O. W. Merten, M. Mezzina, and S. Fisson. Editorial (Thematic Issue: Proceedings from the EMBO Workshop: “Modern DNA Concepts and Tools for Safe Gene Transfer and Modificatio“). Curr Gene Ther, 16(3):153-155, 2016. [PubMed:27457888].
[15] E. Verhoeyen, S. Gomez, A. Galy, E. Ayuso, P. Midoux, M. Puceat, G. Vassaux, and P. Cordelier. Twelfth Annual Meeting of the French Society of Cell and Gene Therapy. Hum. Gene Ther., 27(7):555-558, Jul 2016. [DOI:10.1089/hum.2016.29031.eve] [PubMed:27428658].
[14] T. Aranyi, D. Stockholm, R. Yao, C. Poinsignon, T. Wiart, G. Corre, N. Touleimat, J. Tost, A. Galy, and A. Paldi. Systemic epigenetic response to recombinant lentiviral vectors independent of proviral integration. Epigenetics Chromatin, 9:29, 2016. [PubMed Central:PMC4940770] [DOI:10.1186/s13072-016-0077-1] [PubMed:27408621].
[13] J. Bennett, J. Wellman, K. A. Marshall, S. McCague, M. Ashtari, J. DiStefano-Pappas, O. U. Elci, D. C. Chung, J. Sun, J. F. Wright, D. R. Cross, P. Aravand, L. L. Cyckowski, J. L. Bennicelli, F. Mingozzi, A. Auricchio, E. A. Pierce, J. Ruggiero, B. P. Leroy, F. Simonelli, K. A. High, and A. M. Maguire. 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. Lancet, Jun 2016. [DOI:10.1016/S0140-6736(16)30371-3] [PubMed:27375040].
[12] D. Israeli, J. Poupiot, F. Amor, K. Charton, W. Lostal, L. Jeanson-Leh, and I. Richard. Circulating miRNAs are generic and versatile therapeutic monitoring biomarkers in muscular dystrophies. Sci Rep, 6:28097, 2016. [DOI:10.1038/srep28097] [PubMed:27323895].
[11] N. Holic and D. Fenard. Production of Retrovirus-Based Vectors in Mildly Acidic pH Conditions. Methods Mol. Biol., 1448:41-48, 2016. [DOI:10.1007/978-1-4939-3753-0_3] [PubMed:27317171].
[10] O. Romano, C. Peano, G. M. Tagliazucchi, L. Petiti, V. Poletti, F. Cocchiarella, E. Rizzi, M. Severgnini, A. Cavazza, C. Rossi, P. Pagliaro, A. Ambrosi, G. Ferrari, S. Bicciato, G. De Bellis, F. Mavilio, and A. Miccio. Transcriptional, epigenetic and retroviral signatures identify regulatory regions involved in hematopoietic lineage commitment. Sci Rep, 6:24724, 2016. [PubMed Central:PMC4837375] [DOI:10.1038/srep24724] [PubMed:27095295].
[9] R. Mansour, S. Severin, J. M. Xuereb, M. P. Gratacap, J. Laporte, A. Buj-Bello, H. Tronchere, and B. Payrastre. Expression of myotubularins in blood platelets: Characterization and potential diagnostic of X-linked myotubular myopathy. Biochem. Biophys. Res. Commun., 476(3):167-173, Jul 2016. [DOI:10.1016/j.bbrc.2016.04.127] [PubMed:27155155].
[8] A. Cavazza, A. Miccio, O. Romano, L. Petiti, G. Malagoli Tagliazucchi, C. Peano, M. Severgnini, E. Rizzi, G. De Bellis, S. Bicciato, and F. Mavilio. Dynamic Transcriptional and Epigenetic Regulation of Human Epidermal Keratinocyte Differentiation. Stem Cell Reports, 6(4):618-632, Apr 2016. [PubMed Central:PMC4834057] [DOI:10.1016/j.stemcr.2016.03.003] [PubMed:27050947].
[7] M. Costopoulos, V. Touitou, J. L. Golmard, A. Darugar, S. Fisson, P. Bonnemye, M. L. Le Lez, C. Soussain, N. Cassoux, T. Lamy, P. Le Hoang, B. Bodaghi, H. Merle-Beral, and M. Le Garff-Tavernier. ISOLD: A New Highly Sensitive Interleukin Score for Intraocular Lymphoma Diagnosis. Ophthalmology, Mar 2016. [DOI:10.1016/j.ophtha.2016.01.037] [PubMed:26948307].
[6] I. Richard, J. Y. Hogrel, D. Stockholm, C. A. Payan, F. Fougerousse, B. Eymard, C. Mignard, A. Lopez de Munain, M. Fardeau, and J. A. Urtizberea. Natural history of LGMD2A for delineating outcome measures in clinical trials. Ann Clin Transl Neurol, 3(4):248-265, Apr 2016. [PubMed Central:PMC4818744] [DOI:10.1002/acn3.287] [PubMed:27081656].
[5] S. Gregoire, C. Terrada, G. H. Martin, G. Fourcade, A. Baeyens, G. Marodon, S. Fisson, F. Billiard, B. Lucas, R. Tadayoni, F. Behar-Cohen, B. Levacher, A. Galy, P. LeHoang, D. Klatzmann, B. Bodaghi, and B. L. Salomon. Treatment of Uveitis by In Situ Administration of Ex Vivo-Activated Polyclonal Regulatory T Cells. J. Immunol., 196(5):2109-2118, Mar 2016. [DOI:10.4049/jimmunol.1501723] [PubMed:26826251].
[4] G. Corre, M. Dessainte, J. B. Marteau, B. Dalle, D. Fenard, and A. Galy. "RCL-Pooling Assay": A Simplified Method for the Detection of Replication-Competent Lentiviruses in Vector Batches Using Sequential Pooling. Hum. Gene Ther., 27(2):202-210, Feb 2016. [DOI:10.1089/hum.2015.166] [PubMed:26886834].
[3] P. Rivera-Munoz, V. Abramowski, S. Jacquot, P. Andre, S. Charrier, K. Lipson-Ruffert, A. Fischer, A. Galy, M. Cavazzana, and J. P. de Villartay. Lymphopoiesis in transgenic mice over-expressing Artemis. Gene Ther., 23(2):176-186, Feb 2016. [DOI:10.1038/gt.2015.95] [PubMed:26361272].
[2] S. Majdoul, A. K. Seye, A. Kichler, N. Holic, A. Galy, B. Bechinger, and D. Fenard. Molecular Determinants of Vectofusin-1 and Its Derivatives for the Enhancement of Lentivirally Mediated Gene Transfer into Hematopoietic Stem/Progenitor Cells. J. Biol. Chem., 291(5):2161-2169, Jan 2016. [PubMed Central:PMC4732202] [DOI:10.1074/jbc.M115.675033] [PubMed:26668323].
[1] M. W. Lawlor, A. H. Beggs, A. Buj-Bello, M. K. Childers, J. J. Dowling, E. S. James, H. Meng, S. A. Moore, S. Prasad, B. Schoser, and C. A. Sewry. Skeletal Muscle Pathology in X-Linked Myotubular Myopathy: Review With Cross-Species Comparisons. J. Neuropathol. Exp. Neurol., Jan 2016. [DOI:10.1093/jnen/nlv020] [PubMed:26823526].

2015

[29] Richard, I., Hogrel, J-Y., Stockholm, D., Payan, C.A.M., Fougerousse, F., The calpainopathy study group, Eymard, B., Mignard, C., Lopez de Munain, A., Fardeau, M., Urtizberea, J-A. Natural history of LGMD2A indicates the need to select gender, mutation type and muscles to delineate the best outcome measures for clinical trials. Ann. Clin. Transl. Neurol.on press
[28] A. Galy, G. Corre, M. Cavazzana, and S. Hacein-Bey-Abina. Efficacy and safety of gene therapy for Wiskott-Aldrich syndrome. Med Sci (Paris), 31(12):1066-1069, Dec 2015. [DOI:10.1051/medsci/20153112006] [PubMed:26672655].
[27] S. Majdoul, A. K. Seye, A. Kichler, N. Holic, A. Galy, B. Bechinger, and D. Fenard. Molecular determinants of Vectofusin-1 and its derivatives for the enhancement of lentiviral-mediated gene transfer into hematopoietic stem/progenitor cells. J. Biol. Chem., Dec 2015. [DOI:10.1074/jbc.M115.675033] [PubMed:26668323].
[26] J. Cosette, A. Moussy, F. Onodi, A. Auffret-Cariou, T. M. Neildez-Nguyen, A. Paldi, and D. Stockholm. Single Cell Dynamics Causes Pareto-Like Effect in Stimulated T Cell Populations. Sci Rep, 5:17756, 2015. [PubMed Central:PMC4673432] [DOI:10.1038/srep17756] [PubMed:26648396].
[25] M. K. Childers, A. H. Beggs, and A. Buj-Bello. Gene replacement rescues severe muscle pathology and prolongs survival in myotubularin-deficient mice and dogs. Ann Transl Med, 3(17):257, Oct 2015. [PubMed Central:PMC4620094] [DOI:10.3978/j.issn.2305-5839.2015.10.01] [PubMed:26605303].
[24] R. De Cid, R. Ben Yaou, C. Roudaut, K. Charton, S. Baulande, F. Leturcq, N. B. Romero, E. Malfatti, M. Beuvin, A. Vihola, A. Criqui, I. Nelson, J. Nectoux, L. Ben Aim, C. Caloustian, R. Olaso, B. Udd, G. Bonne, B. Eymard, and I. Richard. A new titinopathy: Childhood-juvenile onset Emery-Dreifuss-like phenotype without cardiomyopathy. Neurology, 85(24):2126-2135, Dec 2015. [DOI:10.1212/WNL.0000000000002200] [PubMed:26581302].
[23] D. J. Hui, S. C. Edmonson, G. M. Podsakoff, G. C. Pien, L. Ivanciu, R. M. Camire, H. Ertl, F. Mingozzi, K. A. High, and E. Basner-Tschakarjan. AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes. Mol Ther Methods Clin Dev, 2:15029, 2015. [PubMed Central:PMC4588448] [DOI:10.1038/mtm.2015.29] [PubMed:26445723].
[22] F. J. Molina-Estevez, A. Nowrouzi, M. Lozano, A. Galy, S. Charrier, C. V. Kalle, G. Guenechea, J. A. Bueren, and M. Schmidt. Lentiviral-Mediated Gene Therapy in Fanconi Anemia-A Mice Reveals Long-Term Engraftment and Continuous Turnover of Corrected HSCs. Curr Gene Ther, 15(6):550-562, 2015. [PubMed:26415575].
[21] P. Rivera-Munoz, V. Abramowski, S. Jacquot, P. Andre, S. Charrier, K. Lipson-Ruffert, A. Fischer, A. Galy, M. Cavazzana, and J. P. de Villartay. Lymphopoiesis in transgenic mice over-expressing Artemis. Gene Ther., Oct 2015. [DOI:10.1038/gt.2015.95] [PubMed:26361272].
[20] F. Boisgerault and F. Mingozzi. The Skeletal Muscle Environment and Its Role in Immunity and Tolerance to AAV Vector-Mediated Gene Transfer. Curr Gene Ther, 15(4):381-394, 2015. [PubMed Central:PMC4515578] [PubMed:26122097].
[19] C. J. Aalbers, L. Bevaart, S. Loiler, K. de Cortie, J. F. Wright, F. Mingozzi, P. P. Tak, and M. J. Vervoordeldonk. Preclinical Potency and Biodistribution Studies of an AAV 5 Vector Expressing Human Interferon-β (ART-I02) for Local Treatment of Patients with Rheumatoid Arthritis. PLoS ONE, 10(6):e0130612, 2015. [PubMed Central:PMC4479517] [DOI:10.1371/journal.pone.0130612] [PubMed:26107769].
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[14] M. Ferrand, S. Da Rocha, G. Corre, A. Galy, and F. Boisgerault. Serotype-specific Binding Properties and Nanoparticle Characteristics Contribute to the Immunogenicity of rAAV1 Vectors. Mol. Ther., 23(6):1022-1033, Jun 2015. [DOI:10.1038/mt.2015.59] [PubMed:25881000].
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[12] F. Mingozzi and H. Buning. Adeno-Associated Viral Vectors at the Frontier between Tolerance and Immunity. Front Immunol, 6:120, 2015. [PubMed Central:PMC4362342] [DOI:10.3389/fimmu.2015.00120] [PubMed:25852689].
[11] A. Meliani, C. Leborgne, S. Triffault, L. Jeanson-Leh, P. Veron, and F. Mingozzi. Determination of anti-adeno-associated virus vector neutralizing antibody titer with an in vitro reporter system. Hum Gene Ther Methods, 26(2):45-53, Apr 2015. [PubMed Central:PMC4403012] [DOI:10.1089/hgtb.2015.037] [PubMed:25819687].
[10] B. Marini, A. Kertesz-Farkas, H. Ali, B. Lucic, K. Lisek, L. Manganaro, S. Pongor, R. Luzzati, A. Recchia, F. Mavilio, M. Giacca, and M. Lusic. Nuclear architecture dictates HIV-1 integration site selection. Nature, 521(7551):227-231, May 2015. [DOI:10.1038/nature14226] [PubMed:25731161].
[9] S. Droz-Georget Lathion, A. Rochat, G. Knott, A. Recchia, D. Martinet, S. Benmohammed, N. Grasset, A. Zaffalon, N. Besuchet Schmutz, E. Savioz-Dayer, J. S. Beckmann, J. Rougemont, F. Mavilio, and Y. Barrandon. A single epidermal stem cell strategy for safe ex vivo gene therapy. EMBO Mol Med, 7(4):380-393, Apr 2015. [PubMed Central:PMC4403041] [DOI:10.15252/emmm.201404353] [PubMed:25724200].
[8] W. Mauhin, O. Lidove, E. Masat, F. Mingozzi, K. Mariampillai, J. M. Ziza, and O. Benveniste. Innate and Adaptive Immune Response in Fabry Disease. JIMD Rep, 22:1-10, 2015. [PubMed Central:PMC4486269] [DOI:10.1007/8904_2014_371] [PubMed:25690728].
[7] T. R. Cheever, D. Berkley, S. Braun, R. H. Brown, B. J. Byrne, J. S. Chamberlain, V. Cwik, D. Duan, H. J. Federoff, K. A. High, B. K. Kaspar, K. W. Klinger, J. Larkindale, J. Lincecum, F. Mavilio, C. L. McDonald, J. McLaughlin, B. Weiss McLeod, J. R. Mendell, G. Nuckolls, H. H. Stedman, D. A. Tagle, L. H. Vandenberghe, H. Wang, P. J. Wernett, J. M. Wilson, J. D. Porter, and A. K. Gubitz. Perspectives on best practices for gene therapy programs. Hum. Gene Ther., 26(3):127-133, Mar 2015. [PubMed Central:PMC4367233] [DOI:10.1089/hum.2014.147] [PubMed:25654329].
[6] N. Junge, F. Mingozzi, M. Ott, and U. Baumann. Adeno-associated virus vector-based gene therapy for monogenetic metabolic diseases of the liver. J. Pediatr. Gastroenterol. Nutr., 60(4):433-440, Apr 2015. [DOI:10.1097/MPG.0000000000000703] [PubMed:25594875].
[5] J. M. Crudele, J. D. Finn, J. I. Siner, N. B. Martin, G. P. Niemeyer, S. Zhou, F. Mingozzi, C. D. Lothrop, and V. R. Arruda. AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice. Blood, 125(10):1553-1561, Mar 2015. [PubMed Central:PMC4351503] [DOI:10.1182/blood-2014-07-588194] [PubMed:25568350].
[4] A. Ribera, V. Haurigot, M. Garcia, S. Marco, S. Motas, P. Villacampa, L. Maggioni, X. Leon, M. Molas, V. Sanchez, S. Munoz, C. Leborgne, X. Moll, M. Pumarola, F. Mingozzi, J. Ruberte, S. Anor, and F. Bosch. Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy. Hum. Mol. Genet., 24(7):2078-2095, Apr 2015. [DOI:10.1093/hmg/ddu727] [PubMed:25524704].
[3] S. O. Han, S. Li, E. D. Brooks, E. Masat, C. Leborgne, S. Banugaria, A. Bird, F. Mingozzi, H. Waldmann, and D. Koeberl. Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade. Hum. Gene Ther., 26(1):26-35, Jan 2015. [PubMed Central:PMC4303018] [DOI:10.1089/hum.2014.115] [PubMed:25382056].
[2] J. Nectoux, R. de Cid, S. Baulande, F. Leturcq, J. A. Urtizberea, I. Penisson-Besnier, A. Nadaj-Pakleza, C. Roudaut, A. Criqui, L. Orhant, D. Peyroulan, R. Ben Yaou, I. Nelson, A. M. Cobo, M. C. Arne-Bes, E. Uro-Coste, P. Nitschke, M. Claustres, G. Bonne, N. Levy, J. Chelly, I. Richard, and M. Cossee. Detection of TRIM32 deletions in LGMD patients analyzed by a combined strategy of CGH array and massively parallel sequencing. Eur. J. Hum. Genet., 23(7):929-934, Jul 2015. [PubMed Central:PMC4463509] [DOI:10.1038/ejhg.2014.223] [PubMed:25351777].
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2014

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[11] T. M. Neildez-Nguyen, J. Bigot, S. Da Rocha, G. Corre, F. Boisgerault, A. Paldi, and A. Galy. Hypoxic culture conditions enhance the generation of regulatory T cells. Immunology, Sep 2014. [DOI:10.1111/imm.12388] [PubMed:25243909].
[10] N. Holic, A. K. Seye, S. Majdoul, S. Martin, O. W. Merten, A. Galy, and D. Fenard. Influence of mildly acidic pH conditions on the production of lentiviral and retroviral vectors. Hum Gene Ther Clin Dev, 25(3):178-185, Sep 2014. [DOI:10.1089/humc.2014.027] [PubMed:25073060].
[9] M. Ferrand, A. Galy, and F. Boisgerault. A dystrophic muscle broadens the contribution and activation of immune cells reacting to rAAV gene transfer. Gene Ther., 21(9):828-839, Sep 2014. [PubMed Central:PMC4283385] [DOI:10.1038/gt.2014.61] [PubMed:25030611].
[8] M. W. Lawlor, M. G. Viola, H. Meng, R. V. Edelstein, F. Liu, K. Yan, E. J. Luna, A. Lerch-Gaggl, R. G. Hoffmann, C. R. Pierson, A. Buj-Bello, J. L. Lachey, S. Pearsall, L. Yang, C. J. Hillard, and A. H. Beggs. Differential muscle hypertrophy is associated with satellite cell numbers and Akt pathway activation following activin type IIB receptor inhibition in Mtm1 p.R69C mice. Am. J. Pathol., 184(6):1831-1842, Jun 2014. [PubMed Central:PMC4044712] [DOI:10.1016/j.ajpath.2014.03.003] [PubMed:24726641].
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[1] M. K. Childers, R. Joubert, K. Poulard, C. Moal, R. W. Grange, J. A. Doering, M. W. Lawlor, B. E. Rider, T. Jamet, N. Daniele, S. Martin, C. Riviere, T. Soker, C. Hammer, L. Van Wittenberghe, M. Lockard, X. Guan, M. Goddard, E. Mitchell, J. Barber, J. K. Williams, D. L. Mack, M. E. Furth, A. Vignaud, C. Masurier, F. Mavilio, P. Moullier, A. H. Beggs, and A. Buj-Bello. Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy. Sci Transl Med, 6(220):220ra10, Jan 2014. [PubMed Central:PMC4105197] [DOI:10.1126/scitranslmed.3007523] [PubMed:24452262].
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