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2019

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[25] P. Colella and F. Mingozzi. Gene Therapy for Pompe Disease: The Time is now. Hum. Gene Ther., 30(10):1245-1262, 10 2019. [DOI:10.1089/hum.2019.109] [PubMed:31298581].
[24] P. Colella, P. Sellier, H. Costa Verdera, F. Puzzo, L. van Wittenberghe, N. Guerchet, N. Daniele, B. Gjata, S. Marmier, S. Charles, M. Simon Sola, I. Ragone, C. Leborgne, F. Collaud, and F. Mingozzi. AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice. Mol Ther Methods Clin Dev, 12:85-101, Mar 2019. [PubMed Central:PMC6299151] [DOI:10.1016/j.omtm.2018.11.002] [PubMed:9668092].
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[19] S. Le Quellec, A. P. Dane, E. Barbon, J. C. Bordet, F. Mingozzi, Y. Dargaud, T. Marais, M. G. Biferi, C. N?grier, A. C. Nathawani, and N. Enjolras. Recombinant Adeno-Associated Viral Vectors Expressing Human Coagulation FIX-E456H Variant in Hemophilia B Mice. Thromb. Haemost., 119(12):1956-1967, Dec 2019. [DOI:10.1055/s-0039-1697658] [PubMed:31659733].
[18] J. Poupiot, H. Costa Verdera, R. Hardet, P. Colella, F. Collaud, L. Bartolo, J. Davoust, P. Sanatine, F. Mingozzi, I. Richard, and G. Ronzitti. Role of Regulatory T Cell and Effector T Cell Exhaustion in Liver-Mediated Transgene Tolerance in Muscle. Mol Ther Methods Clin Dev, 15:83-100, Dec 2019. [PubMed Central:PMC6804827] [DOI:10.1016/j.omtm.2019.08.012] [PubMed:28284982].
[17] C. Radek, O. Bernadin, K. Drechsel, N. Cordes, R. Pfeifer, P. Str??er, M. Mormin, A. Gutierrez-Guerrero, F. L. Cosset, A. D. Kaiser, T. Schaser, A. Galy, E. Verhoeyen, and I. C. D. Johnston. Vectofusin-1 Improves Transduction of Primary Human Cells with Diverse Retroviral and Lentiviral Pseudotypes, Enabling Robust, Automated Closed-System Manufacturing. Hum. Gene Ther., 30(12):1477-1493, 12 2019. [PubMed Central:PMC6919281] [DOI:10.1089/hum.2019.157] [PubMed:30224381].
[16] P. R?o, S. Navarro, W. Wang, R. S?nchez-Dom?nguez, R. M. Pujol, J. C. Segovia, M. Bogliolo, E. Merino, N. Wu, R. Salgado, M. L. Lamana, R. M. Ya?ez, J. A. Casado, Y. Gim?nez, F. J. Rom?n-Rodr?guez, L. ?lvarez, O. Alberquilla, A. Raimbault, G. Guenechea, M. L. Lozano, L. Cerrato, M. Hernando, E. G?lvez, R. Hladun, I. Giralt, J. Barquinero, A. Galy, N. Garc?a de Ando?n, R. L?pez, A. Catal?, J. D. Schwartz, J. Surrall?s, J. Soulier, M. Schmidt, C. D?az de Heredia, J. Sevilla, and J. A. Bueren. Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia. Nat. Med., 25(9):1396-1401, 09 2019. [DOI:10.1038/s41591-019-0550-z] [PubMed:31501599].
[15] N. S. Orefice, B. Souchet, J. Braudeau, S. Alves, F. Piguet, F. Collaud, G. Ronzitti, S. Tada, P. Hantraye, F. Mingozzi, F. Ducong?, and N. Cartier. Real-Time Monitoring of Exosome Enveloped-AAV Spreading by Endomicroscopy Approach: A New Tool for Gene Delivery in the Brain. Mol Ther Methods Clin Dev, 14:237-251, Sep 2019. [PubMed Central:PMC6699252] [DOI:10.1016/j.omtm.2019.06.005] [PubMed:9813665].
[14] G. Ronzitti, F. Collaud, P. Laforet, and F. Mingozzi. Progress and challenges of gene therapy for Pompe disease. Ann Transl Med, 7(13):287, Jul 2019. [PubMed Central:PMC6642941] [DOI:10.21037/atm.2019.04.67] [PubMed:9829534].
[13] L. Jauze, L. Monteillet, G. Mithieux, F. Rajas, and G. Ronzitti. Challenges of Gene Therapy for the Treatment of Glycogen Storage Diseases Type I and Type III. Hum. Gene Ther., 30(10):1263-1273, 10 2019. [DOI:10.1089/hum.2019.102] [PubMed:31319709].
[12] E. Casajus Pelegay, F. Puzzo, A. Yilmazer, and U. Cagin. Targeting Mitochondrial Defects to Increase Longevity in Animal Models of Neurodegenerative Diseases. Adv. Exp. Med. Biol., 1134:89-110, 2019. [DOI:10.1007/978-3-030-12668-1_5] [PubMed:30919333].
[11] M. Lo Scrudato, K. Poulard, C. Sourd, S. Tom?, A. F. Klein, G. Corre, A. Huguet, D. Furling, G. Gourdon, and A. Buj-Bello. Genome Editing of Expanded CTG Repeats within the Human DMPK Gene Reduces Nuclear RNA Foci in the Muscle of DM1 Mice. Mol. Ther., 27(8):1372-1388, 08 2019. [PubMed Central:PMC6697452] [DOI:10.1016/j.ymthe.2019.05.021] [PubMed:29056323].
[10] D. W. Scott and F. Mingozzi. Balancing immunity and tolerance in gene therapy for inherited and acquired diseases. Cell. Immunol., 342:103945, 08 2019. [DOI:10.1016/j.cellimm.2019.103945] [PubMed:31229195].
[9] D. Israeli, J. Cosette, G. Corre, F. Amor, J. Poupiot, D. Stockholm, M. Montus, B. Gjata, and I. Richard. An AAV-SGCG Dose-Response Study in a γ-Sarcoglycanopathy Mouse Model in the Context of Mechanical Stress. Mol Ther Methods Clin Dev, 13:494-502, Jun 2019. [PubMed Central:PMC6545357] [DOI:10.1016/j.omtm.2019.04.007] [PubMed:9732288].
[8] L. Hoch, S. F. Henriques, C. Bruge, J. Marsolier, M. Benabides, N. Bourg, J. Tournois, G. Mah?, L. Morizur, M. Jarrige, A. Bigot, I. Richard, and X. Nissan. Identification of thiostrepton as a pharmacological approach to rescue misfolded alpha-sarcoglycan mutant proteins from degradation. Sci Rep, 9(1):6915, 05 2019. [PubMed Central:PMC6502821] [DOI:10.1038/s41598-019-43399-w] [PubMed:31061434].
[7] C. Kutchukian, P. Szentesi, B. Allard, A. Buj-Bello, L. Csernoch, and V. Jacquemond. Ca2+-induced sarcoplasmic reticulum Ca2+ release in myotubularin-deficient muscle fibers. Cell Calcium, 80:91-100, 06 2019. [DOI:10.1016/j.ceca.2019.04.004] [PubMed:30999217].
[6] W. Lostal, D. Stockholm, and I. Richard. In Vivo Monitoring of Calpain Activity by Forster Resonance Energy Transfer. Methods Mol. Biol., 1915:57-66, 2019. [DOI:10.1007/978-1-4939-8988-1_5] [PubMed:30617795].
[5] A. Lattanzi, V. Meneghini, G. Pavani, F. Amor, S. Ramadier, T. Felix, C. Antoniani, C. Masson, O. Alibeu, C. Lee, M. H. Porteus, G. Bao, M. Amendola, F. Mavilio, and A. Miccio. Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements. Mol. Ther., 27(1):137-150, 01 2019. [PubMed Central:PMC6318785] [DOI:10.1016/j.ymthe.2018.10.008] [PubMed:25733580].
[4] C. Leborgne, V. Latournerie, S. Boutin, D. Desgue, A. Qu?r?, E. Pignot, F. Collaud, S. Charles, M. Simon Sola, E. Masat, F. Jouen, O. Boyer, C. Masurier, F. Mingozzi, and P. Veron. Prevalence and long-term monitoring of humoral immunity against adeno-associated virus in Duchenne Muscular Dystrophy patients. Cell. Immunol., 342:103780, 08 2019. [DOI:10.1016/j.cellimm.2018.03.004] [PubMed:29571923].
[3] W. Lostal, C. Roudaut, M. Faivre, K. Charton, L. Suel, N. Bourg, H. Best, J. E. Smith, J. Gohlke, G. Corre, X. Li, Z. Elbeck, R. Kn?ll, J. Y. Deschamps, H. Granzier, and I. Richard. Titin splicing regulates cardiotoxicity associated with calpain 3 gene therapy for limb-girdle muscular dystrophy type 2A. Sci Transl Med, 11(520), 11 2019. [DOI:10.1126/scitranslmed.aat6072] [PubMed:31776291].
[2] R. Cabrera-P?rez, F. Vila-Juli?, M. Hirano, F. Mingozzi, J. Torres-Torronteras, and R. Mart? Alpha-1-Antitrypsin Promoter Improves the Efficacy of an Adeno-Associated Virus Vector for the Treatment of Mitochondrial Neurogastrointestinal Encephalomyopathy. Hum. Gene Ther., 30(8):985-998, 08 2019. [DOI:10.1089/hum.2018.217] [PubMed:30900470].
[1] E. Danese, S. Raimondi, M. Montagnana, A. Tagetti, T. Langaee, P. Borgiani, C. Ciccacci, A. J. Carcas, A. M. Borobia, H. Y. Tong, C. D?vila-Fajardo, M. Rodrigues Botton, S. Bourgeois, P. Deloukas, M. D. Caldwell, J. K. Burmester, R. L. Berg, L. H. Cavallari, K. Drozda, M. Huang, L. Z. Zhao, H. J. Cen, R. Gonzalez-Conejero, V. Roldan, Y. Nakamura, T. Mushiroda, I. Y. Gong, R. B. Kim, K. Hirai, K. Itoh, C. Isaza, L. Beltr?n, E. Jim?nez-Varo, M. Ca?adas-Garre, A. Giontella, M. K. Kringen, K. B. F. Haug, H. S. Gwak, K. E. Lee, P. Minuz, M. T. M. Lee, S. A. Lubitz, S. Scott, C. Mazzaccara, L. Sacchetti, E. Gen?, M. ?zer, A. Pathare, R. Krishnamoorthy, A. Paldi, V. Siguret, M. A. Loriot, V. K. Kutala, G. Suarez-Kurtz, J. Perini, J. C. Denny, A. H. Ramirez, B. Mittal, S. S. Rathore, H. Sagreiya, R. Altman, M. H. A. Shahin, S. I. Khalifa, N. A. Limdi, C. Rivers, A. Shendre, C. Dillon, I. M. Suriapranata, H. H. Zhou, S. L. Tan, V. Tatarunas, V. Lesauskaite, Y. Zhang, A. H. Maitland-van der Zee, T. I. Verhoef, A. de Boer, M. Taljaard, C. F. Zambon, V. Pengo, J. E. Zhang, M. Pirmohamed, J. A. Johnson, and C. Fava. Effect of CYP4F2, VKORC1, and CYP2C9 in Influencing Coumarin Dose: A Single-Patient Data Meta-Analysis in More Than 15,000 Individuals. Clin. Pharmacol. Ther., 105(6):1477-1491, 06 2019. [PubMed Central:PMC6542461] [DOI:10.1002/cpt.1323] [PubMed:29238065].

2018

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2017

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[4] 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, 04 2017. [PubMed Central:PMC5383631] [DOI:10.1016/j.ymthe.2017.02.004] [PubMed:28237839].
[3] R. Hardet and F. Mingozzi. Oral Tolerance: Another Reason to Eat Your Veggies! Mol. Ther., 25(2):311-313, 02 2017. [PubMed Central:PMC5368840] [DOI:10.1016/j.ymthe.2017.01.001] [PubMed:28109961].
[2] 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, 04 2017. [DOI:10.1089/hgtb.2016.150] [PubMed:28042946].
[1] 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, 56(1):129-135, Jul 2017. [DOI:10.1002/mus.25443] [PubMed:27759885].

2016

[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].
[18] J. Rouillon, J. Poupiot, A. Zocevic, F. Amor, T. Leger, C. Garcia, J. M. Camadro, B. Wong, R. Pinilla, J. Cosette, A. M. Coenen-Stass, G. Mcclorey, T. C. Roberts, M. J. Wood, L. Servais, B. Udd, T. Voit, I. Richard, and F. Svinartchouk. Serum proteomic profiling reveals fragments of MYOM3 as potential biomarkers for monitoring the outcome of therapeutic interventions in muscular dystrophies. Hum. Mol. Genet., 24(17):4916-4932, Sep 2015. [PubMed Central:PMC4527491] [DOI:10.1093/hmg/ddv214] [PubMed:26060189].
[17] M. Pryadkina, W. Lostal, N. Bourg, K. Charton, C. Roudaut, M. L. Hirsch, and I. Richard. A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequence. Mol Ther Methods Clin Dev, 2:15009, 2015. [PubMed Central:PMC4445010] [DOI:10.1038/mtm.2015.9] [PubMed:26029720].
[16] V. Poletti, A. Delli Carri, G. Malagoli Tagliazucchi, A. Faedo, L. Petiti, E. M. Mazza, C. Peano, G. De Bellis, S. Bicciato, A. Miccio, E. Cattaneo, and F. Mavilio. Genome-Wide Definition of Promoter and Enhancer Usage during Neural Induction of Human Embryonic Stem Cells. PLoS ONE, 10(5):e0126590, 2015. [PubMed Central:PMC4433211] [DOI:10.1371/journal.pone.0126590] [PubMed:25978676].
[15] S. Hacein-Bey Abina, H. B. Gaspar, J. Blondeau, L. Caccavelli, S. Charrier, K. Buckland, C. Picard, E. Six, N. Himoudi, K. Gilmour, A. M. McNicol, H. Hara, J. Xu-Bayford, C. Rivat, F. Touzot, F. Mavilio, A. Lim, J. M. Treluyer, S. Heritier, F. Lefrere, J. Magalon, I. Pengue-Koyi, G. Honnet, S. Blanche, E. A. Sherman, F. Male, C. Berry, N. Malani, F. D. Bushman, A. Fischer, A. J. Thrasher, A. Galy, and M. Cavazzana. Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome. JAMA, 313(15):1550-1563, Apr 2015. [DOI:10.1001/jama.2015.3253] [PubMed:25898053].
[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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2014

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