2018

[11] J. Vendomele, S. Dehmani, Q. Khebizi, A. Galy, and S. Fisson. Subretinal Injection of HY Peptides Induces Systemic Antigen-Specific Inhibition of Effector CD4+ and CD8+ T-Cell Responses. Front Immunol, 9:504, 2018. [PubMed Central:PMC5890180] [DOI:10.3389/fimmu.2018.00504] [PubMed:29662488].
[10] M. Holstein, C. Mesa-Nunez, C. Miskey, E. Almarza, V. Poletti, M. Schmeer, E. Grueso, J. C. Ordonez Flores, D. Kobelt, W. Walther, M. K. Aneja, J. Geiger, H. B. Bonig, Z. Izsvak, M. Schleef, C. Rudolph, F. Mavilio, J. A. Bueren, G. Guenechea, and Z. Ivics. Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors. Mol. Ther., 26(4):1137-1153, Apr 2018. [DOI:10.1016/j.ymthe.2018.01.012] [PubMed:29503198].
[9] P. H. Jonson, J. Palmio, M. Johari, S. Penttila, A. Evila, I. Nelson, G. Bonne, N. Wiart, V. Meyer, A. Boland, J. F. Deleuze, C. Masson, T. Stojkovic, F. Chapon, N. B. Romero, G. Sole, X. Ferrer, A. Ferreiro, P. Hackman, I. Richard, and B. Udd. Novel mutations in DNAJB6 cause LGMD1D and distal myopathy in French families. Eur. J. Neurol., 25(5):790-794, May 2018. [DOI:10.1111/ene.13598] [PubMed:29437287].
[8] N. Daniele, C. Moal, L. Julien, M. Marinello, T. Jamet, S. Martin, A. Vignaud, M. W. Lawlor, and A. Buj-Bello. Intravenous Administration of a MTMR2-Encoding AAV Vector Ameliorates the Phenotype of Myotubular Myopathy in Mice. J. Neuropathol. Exp. Neurol., 77(4):282-295, Apr 2018. [DOI:10.1093/jnen/nly002] [PubMed:29408998].
[7] S. F. Henriques, C. Patissier, N. Bourg, C. Fecchio, D. Sandona, J. Marsolier, and I. Richard. Different outcome of sarcoglycan missense mutation between human and mouse. PLoS ONE, 13(1):e0191274, 2018. [PubMed Central:PMC5779665] [DOI:10.1371/journal.pone.0191274] [PubMed:29360879].
[6] M. Carotti, J. Marsolier, M. Soardi, E. Bianchini, C. Gomiero, C. Fecchio, S. F. Henriques, R. Betto, R. Sacchetto, I. Richard, and D. Sandona. Repairing folding-defective α-sarcoglycan mutants by CFTR correctors, a potential therapy for limb-girdle muscular dystrophy 2D. Hum. Mol. Genet., 27(6):969-984, Mar 2018. [PubMed Central:PMC5886177] [DOI:10.1093/hmg/ddy013] [PubMed:29351619].
[5] P. Colella, G. Ronzitti, and F. Mingozzi. Emerging Issues in AAV-Mediated In Vivo Gene Therapy. Mol Ther Methods Clin Dev, 8:87-104, Mar 2018. [PubMed Central:PMC5758940] [DOI:10.1016/j.omtm.2017.11.007] [PubMed:29326962].
[4] G. Ronzitti and F. Mingozzi. Combination Therapy Is the New Gene Therapy? Mol. Ther., 26(1):12-14, Jan 2018. [PubMed Central:PMC5763149] [DOI:10.1016/j.ymthe.2017.12.008] [PubMed:29273499].
[3] V. Poletti and F. Mavilio. Interactions between Retroviruses and the Host Cell Genome. Mol Ther Methods Clin Dev, 8:31-41, Mar 2018. [PubMed Central:PMC5684498] [DOI:10.1016/j.omtm.2017.10.001] [PubMed:29159201].
[2] A. Paldi. Conceptual Challenges of the Systemic Approach in Understanding Cell Differentiation. Methods Mol. Biol., 1702:27-39, 2018. [DOI:10.1007/978-1-4939-7456-6_3] [PubMed:29119500].
[1] N. K. Paulk, K. Pekrun, E. Zhu, S. Nygaard, B. Li, J. Xu, K. Chu, C. Leborgne, A. P. Dane, A. Haft, Y. Zhang, F. Zhang, C. Morton, M. B. Valentine, A. M. Davidoff, A. C. Nathwani, F. Mingozzi, M. Grompe, I. E. Alexander, L. Lisowski, and M. A. Kay. Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity. Mol. Ther., 26(1):289-303, Jan 2018. [PubMed Central:PMC5763027] [DOI:10.1016/j.ymthe.2017.09.021] [PubMed:29055620].

2017

[35] A. Boucheham, V. Sommard, F. Zehraoui, A. Boualem, M. Batouche, A. Bendahmane, D. Israeli, and F. Tahi. IpiRId: Integrative approach for piRNA prediction using genomic and epigenomic data. PLoS ONE, 12(6):e0179787, 2017. [PubMed Central:PMC5473586] [DOI:10.1371/journal.pone.0179787] [PubMed:28622364].
[34] T. Soheili, A. Durand, F. E. Sepulveda, J. Riviere, C. Lagresle-Peyrou, H. Sadek, G. de Saint Basile, S. Martin, F. Mavilio, M. Cavazzana, and I. Andre-Schmutz. Gene transfer into hematopoietic stem cells reduces HLH manifestations in a murine model of Munc13-4 deficiency. Blood Adv, 1(27):2781-2789, Dec 2017. [PubMed Central:PMC5745141] [DOI:10.1182/bloodadvances.2017012088] [PubMed:29296930].
[33] A. Meliani, F. Boisgerault, Z. Fitzpatrick, S. Marmier, C. Leborgne, F. Collaud, M. Simon Sola, S. Charles, G. Ronzitti, A. Vignaud, L. van Wittenberghe, B. Marolleau, F. Jouen, S. Tan, O. Boyer, O. Christophe, A. R. Brisson, C. A. Maguire, and F. Mingozzi. Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors. Blood Adv, 1(23):2019-2031, Oct 2017. [PubMed Central:PMC5728288] [DOI:10.1182/bloodadvances.2017010181] [PubMed:29296848].
[32] J. Vendomele, Q. Khebizi, and S. Fisson. Cellular and Molecular Mechanisms of Anterior Chamber-Associated Immune Deviation (ACAID): What We Have Learned from Knockout Mice. Front Immunol, 8:1686, 2017. [PubMed Central:PMC5714853] [DOI:10.3389/fimmu.2017.01686] [PubMed:29250068].
[31] F. Puzzo, P. Colella, M. G. Biferi, D. Bali, N. K. Paulk, P. Vidal, F. Collaud, M. Simon-Sola, S. Charles, R. Hardet, C. Leborgne, A. Meliani, M. Cohen-Tannoudji, S. Astord, B. Gjata, P. Sellier, L. van Wittenberghe, A. Vignaud, F. Boisgerault, M. Barkats, P. Laforet, M. A. Kay, D. D. Koeberl, G. Ronzitti, and F. Mingozzi. Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase. Sci Transl Med, 9(418), Nov 2017. [PubMed Central:PMC5826611] [DOI:10.1126/scitranslmed.aam6375] [PubMed:29187643].
[30] Patrice Vidal, Serena Pagliarani, Pasqualina Colella, Helena Costa Verdera, Louisa Jauze, Monika Gjorgjieva, Francesco Puzzo, Solenne Marmier, Fanny Collaud, Marcelo Simon Sola, et al. Rescue of gsdiii phenotype with gene transfer requires liver-and muscle-targeted gde expression. Molecular Therapy, 2017.
[29] A Vihola, H Luque, M Savarese, S Penttilä, M Lindfors, F Leturcq, B Eymard, G Tasca, B Brais, T Conte, et al. Diagnostic anoctamin-5 protein defect in patients with ano5-mutated muscular dystrophy. Neuropathology and applied neurobiology, 2017.
[28] L. S. Vermeer, L. Hamon, A. Schirer, M. Schoup, J. Cosette, S. Majdoul, D. Pastre, D. Stockholm, N. Holic, P. Hellwig, A. Galy, D. Fenard, and B. Bechinger. Vectofusin-1, a potent peptidic enhancer of viral gene transfer forms pH-dependent α-helical nanofibrils, concentrating viral particles. Acta Biomater, 64:259-268, Dec 2017. [DOI:10.1016/j.actbio.2017.10.009] [PubMed:29017974].
[27] F. Mingozzi and K. A. High. Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape. Annu Rev Virol, 4(1):511-534, Sep 2017. [DOI:10.1146/annurev-virology-101416-041936] [PubMed:28961410].
[26] Virginie Mariot, Romain Joubert, Christophe Hourdé, Léonard Féasson, Michael Hanna, Francesco Muntoni, Thierry Maisonobe, Laurent Servais, Caroline Bogni, Rozen Panse, et al. Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches. Nature communications, 8(1):1859, 2017.
[25] S. Majdoul, J. Cosette, A. K. Seye, E. Bernard, S. Frin, N. Holic, N. Chazal, L. Briant, L. Espert, A. Galy, and D. Fenard. Peptides derived from evolutionarily conserved domains in Beclin-1 and Beclin-2 enhance the entry of lentiviral vectors into human cells. J. Biol. Chem., 292(45):18672-18681, 11 2017. [PubMed Central:PMC5682973] [DOI:10.1074/jbc.M117.800813] [PubMed:28928217].
[24] G. Ferrari, M. Cavazzana, and F. Mavilio. Gene Therapy Approaches to Hemoglobinopathies. Hematol. Oncol. Clin. North Am., 31(5):835-852, 10 2017. [DOI:10.1016/j.hoc.2017.06.010] [PubMed:28895851].
[23] C. Vandamme, O. Adjali, and F. Mingozzi. Unraveling the Complex Story of Immune Responses to AAV Vectors Trial After Trial. Hum. Gene Ther., 28(11):1061-1074, Nov 2017. [PubMed Central:PMC5649404] [DOI:10.1089/hum.2017.150] [PubMed:28835127].
[22] A. Galy. Major Advances in the Development of Vectors for Clinical Gene Therapy of Hematopoietic Stem Cells from European Groups over the Last 25 Years. Hum. Gene Ther., 28(11):964-971, Nov 2017. [DOI:10.1089/hum.2017.152] [PubMed:28806879].
[21] Oriana Romano, Ingrid Cifola, Valentina Poletti, Marco Severgnini, Clelia Peano, Gianluca De Bellis, Fulvio Mavilio, and Annarita Miccio. Retroviral scanning: Mapping mlv integration sites to define cell-specific regulatory regions. Journal of visualized experiments: JoVE, (123), 2017.
[20] P. Rio, S. Navarro, G. Guenechea, R. Sanchez-Dominguez, M. L. Lamana, R. Yanez, J. A. Casado, P. A. Mehta, M. R. Pujol, J. Surralles, S. Charrier, A. Galy, J. C. Segovia, C. Diaz de Heredia, J. Sevilla, and J. A. Bueren. Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD34+ cells from Fanconi anemia patients. Blood, 130(13):1535-1542, 09 2017. [DOI:10.1182/blood-2017-03-774174] [PubMed:28801449].
[19] A. Matet, C. Kostic, A. P. Bemelmans, A. Moulin, S. G. Rosolen, S. Martin, F. Mavilio, V. Amirjanians, K. Stieger, B. Lorenz, F. Behar-Cohen, and Y. Arsenijevic. Evaluation of tolerance to lentiviral LV-RPE65 gene therapy vector after subretinal delivery in non-human primates. Transl Res, 188:40-57, 10 2017. [DOI:10.1016/j.trsl.2017.06.012] [PubMed:28754419].
[18] A. Moussy, J. Cosette, R. Parmentier, C. da Silva, G. Corre, A. Richard, O. Gandrillon, D. Stockholm, and A. Paldi. Integrated time-lapse and single-cell transcription studies highlight the variable and dynamic nature of human hematopoietic cell fate commitment. PLoS Biol., 15(7):e2001867, Jul 2017. [PubMed Central:PMC5531424] [DOI:10.1371/journal.pbio.2001867] [PubMed:28749943].
[17] Caroline Le Guiner, Laurent Servais, Marie Montus, Thibaut Larcher, Bodvaël Fraysse, Sophie Moullec, Marine Allais, Virginie François, Maeva Dutilleul, Alberto Malerba, et al. Long-term microdystrophin gene therapy is effective in a canine model of duchenne muscular dystrophy. Nature communications, 8:16105, 2017.
[16] Gilles Moulay, Christian Leborgne, A James Mason, Christopher Aisenbrey, Antoine Kichler, and Burkhard Bechinger. Histidine-rich designer peptides of the lah4 family promote cell delivery of a multitude of cargo. Journal of Peptide Science, 23(4):320-328, 2017.
[15] E. C. Morris, T. Fox, R. Chakraverty, R. Tendeiro, K. Snell, C. Rivat, S. Grace, K. Gilmour, S. Workman, K. Buckland, K. Butler, R. Chee, A. D. Salama, H. Ibrahim, H. Hara, C. Duret, F. Mavilio, F. Male, F. D. Bushman, A. Galy, S. O. Burns, H. B. Gaspar, and A. J. Thrasher. Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult. Blood, 130(11):1327-1335, 09 2017. [PubMed Central:PMC5813727] [DOI:10.1182/blood-2017-04-777136] [PubMed:28716862].
[14] A. Lattanzi, S. Duguez, A. Moiani, A. Izmiryan, E. Barbon, S. Martin, K. Mamchaoui, V. Mouly, F. Bernardi, F. Mavilio, and M. Bovolenta. Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System. Mol Ther Nucleic Acids, 7:11-19, Jun 2017. [PubMed Central:PMC5363679] [DOI:10.1016/j.omtn.2017.02.004] [PubMed:28624187].
[13] F. Mavilio. Developing gene and cell therapies for rare diseases: an opportunity for synergy between academia and industry. Gene Ther., 24(9):590-592, Sep 2017. [DOI:10.1038/gt.2017.36] [PubMed:28485723].
[12] 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].
[11] 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, 56(5):943-953, Nov 2017. [PubMed Central:PMC5620115] [DOI:10.1002/mus.25658] [PubMed:28370029].
[10] Christian Leborgne, Debborah Alimi-Guez, Nelly El Shafey, Laetitia van Wittenberghe, Pascal Bigey, Daniel Scherman, and Antoine Kichler. The absorption enhancer sodium deoxycholate promotes high gene transfer in skeletal muscles. International journal of pharmaceutics, 523(1):291-299, 2017.
[9] 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].
[8] 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, 05 2017. [DOI:10.1093/hmg/ddx066] [PubMed:28334834].
[7] 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].
[6] 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, 66(1):252-265, 07 2017. [PubMed Central:PMC5518300] [DOI:10.1002/hep.29176] [PubMed:28318036].
[5] 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].
[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].
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[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].
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[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].
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[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

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[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].
[13] K. Charton, J. Sarparanta, A. Vihola, A. Milic, P. H. Jonson, L. Suel, H. Luque, I. Boumela, I. Richard, and B. Udd. CAPN3-mediated processing of C-terminal titin replaced by pathological cleavage in titinopathy. Hum. Mol. Genet., 24(13):3718-3731, Jul 2015. [DOI:10.1093/hmg/ddv116] [PubMed:25877298].
[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].
[1] R. Ben Abdelwahed Bagga, S. Donnou, J. Cosette, C. Sautes-Fridman, M. Aouni, and S. Fisson. Mouse models of primary central nervous system lymphomas: tools for basing funding and therapeutic strategies. J. Neurooncol., 121(1):9-18, Jan 2015. [DOI:10.1007/s11060-014-1624-x] [PubMed:25300908].

2014

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