Scientific publications 2009

1Alimi-Guez, D., Leborgne, C., Pembouong, G., Van Wittenberghe, L., Mignet, N., Scherman, D. and Kichler, A.“Evaluation of the muscle gene transfer activity of a series of amphiphilic triblock copolymers”. J Gene Med Published Online 15 Sep (2009).

2. Andriola Silva, A.K., Richard, C., Bessodes, M., Scherman, D. and Merten, O.-W.: “Growth Factor Delivery Approaches in Hydrogels”. Biomacromolecules 10 (2009) 9-18.

3Bouazza, B., Kratassiouk, G., Gjata, B., Perie, S., Guily, J.L., Butler-Browne, G.S. andSvinartchouk, F.“Analysis of growth factor expression in affected and unaffected muscles of oculo-pharyngeal muscular dystrophy (OPMD) patients: A pilot study”. Neuromuscul Disord 19 (2009) 199-206.

4. Ciron, C., Cressant, A., Roux, F., Raoul, S., Cherel, Y., Hantraye, P., Deglon, N., Schwartz, B.,Barkats, M., Heard, J.M., Tardieu, M., Moullier, P. and Colle, M.A.: “Human alpha-Iduronidase Gene Transfer Mediated by Adeno-Associated Virus Types 1, 2, and 5 in the Brain of Nonhuman Primates: Vector Diffusion and Biodistribution”. Hum Gene Ther 20 (2009) 350-360.

5Denard, J., Rundwasser, S., Laroudie, N., Gonnet, F., Naldini, L., Radrizzani, M., Galy, A., Merten, O.-W., Danos, O. and Svinartchouk, F.: “Quantitative proteomic analysis of lentiviral vectors using two dimensional gel electrophoresis”. Proteomics 9 (2009) 3666-3676.

6Duque, S., Joussemet, B., Riviere, C., Marais, T., Dubreil, L., Douar, A.M., Fyfe, J., Moullier, P., Colle, M.A. and Barkats, M.“Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons”. Mol Ther 17 (2009) 1187–1196.

7. Gérard, A., Ghiotto, M., Fos, C., Guittard, G., Compagno, D., Galy, A., Lemay, S., Olive, D. and Nunès, J.A.: “Dok-4 is a novel negative regulator of T cell activation”. J Immunol 182 (2009) 7681-7689.

8Gerard, X., Vignaud, L., Charkes, S., Pinset, C., Scherman, D., Kichler, A. and Israeli, D.:“Real-time monitoring of cell transplantation in mouse dystrophic muscles by a secreted alkaline phosphatase reporter gene”. Gene Ther 16 (2009) 815–819.

9. Goizet, C., Boukhris, A., Durr, A., Beetz, C., Truchetto, J., Tesson, C., Tsaousidou, M., Forlani, S., Guyant-Marechal, L., Fontaine, B., Guimaraes, J., Isidor, B., Chazouilleres, O., Wendum, D.,Grid, D., Chevy, F., Chinnery, P.F., Coutinho, P., Azulay, J.P., Feki, I., Mochel, F., Wolf, C., Mhiri, C., Crosby, A., Brice, A. and Stevanin, G.: “CYP7B1 mutations in pure and complex forms of hereditary spastic paraplegia type 5”. Brain 132 (2009) 1589-600.

10. Jedidi, A., Marty, C., Oligo, C., Jeanson-Leh, L., Ribeil, J.A., Casadevall, N., Galy, A., Vainchenker, W. and Villeval, J.L.: “Selective reduction of JAK2V617F-dependent cell growth by siRNA / shRNA and its reversal by cytokines”. Blood 114 (2009) 1842-1851.

11Laure, L., Suel, L., Roudaut, C., Bourg, N., Ouali, A., Bartoli, M., Richard, I. and Daniele, N.:“Cardiac ankyrin repeat protein is a marker of skeletal muscle pathological remodelling”. Febs J 276 (2009) 669-84.

12Leclerc, X., Danos, O., Scherman, D. and Kichler, A.: “A comparison of synthetic oligodeoxynucleotides, DNA fragments and AAV-1 for targeted episomal and chromosomal gene repair”. BMC Biotechnol 9 (2009) 35.

13Le Roy, F., Charton, K., Lorson, C.L. and Richard, I.“RNA-targeting approaches for neuromuscular diseases”. Trends Mol Med Available online 10 November (2009).

14. Mantovani, J., Charrier, S., Eckenberg, R., Saurin, W., Danos, O., Perea, J. and Galy, A.:“Diverse genomic integration of a lentiviral vector developed for the treatment of Wiskott-Aldrich syndrome”. J Gene Med 11 (2009) 645 – 654.

15. Marangoni, F., Bosticardo, M., Charrier, S., Draghici, E., Locci, M., Scaramuzza, S., Panaroni, C., Ponzoni, M., Sanvito, F., Doglioni, C., Liabeuf, M., Gjata, B., Montus, M., Siminovitch, K., Aiuti, A., Naldini, L., Dupre, L., Roncarolo, M.G., Galy, A. and Villa, A.: “Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott-Aldrich Syndrome in Preclinical Models”. Mol Ther 17 (2009) 1073–1082.

16. Megarbane, H., Florence, J., Oliver Sass, J., Schwonbeck, S., Foglio, M., de Cid, R., Cure, S.,Saker, S., Megarbane, A. and Fischer, J.: “An Autosomal-Recessive Form of Cutis Laxa Is Due to Homozygous Elastin Mutations, and the Phenotype May Be Modified by a Heterozygous Fibulin 5 Polymorphism”. J Invest Dermatol 129 (2009) 1650–1655.

17. Mellgren, R.L., Miyake, K., Kramerova, I., Spencer, M.J., Bourg, N., Bartoli, M., Richard, I., Greer, P.A. and McNeil, P.L.: “Calcium-dependent plasma membrane repair requires m- or mu-calpain, but not calpain-3, the proteasome, or caspases”. Biochim Biophys Acta Available online 23 September (2009).

18. Modlich, U., Navarro, S., Zychlinski, D., Maetzig, T., Knoess, S., Brugman, M.H., Schambach, A.,Charrier, S., Galy, A., Thrasher, A., Bueren, J. and Baum, C.: “Insertional transformation of hematopoietic cells by selfinactivating lentiviral and gammaretroviral vectors”. Mol Ther (Published online 11 August 2009).

19. Oudot, T., Lesueur, F., Guedj, M., de Cid, R., McGinn, S., Heath, S., Foglio, M., Prum, B., Lathrop, M., Prud’homme, J.F. and Fischer, J.: “An Association Study of 22 Candidate Genes in Psoriasis Families Reveals Shared Genetic Factors with Other Autoimmune and Skin Disorders”. J Invest Dermatol Published online 25 June (2009).

20. Tazir, M., Ali-Pacha, L., M’Zahem, A., Delaunoy, J.P., Fritsch, M., Nouioua, S., Benhassine, T., Assami, S., Grid, D., Vallat, J.M., Hamri, A. and Koenig, M.: “Ataxia with oculomotor apraxia type 2: A clinical and genetic study of 19 patients”. J Neurol Sci 278 (2009) 77-81.

21. Tazir, M., Nouioua, S., Magy, L., Huehne, K., Assami, S., Urtizberea, A., Grid, D., Hamadouche, T., Rautenstrauss, B. and Vallat, J.-M.: “Phenotypic variability in giant axonal neuropathy”. Neuromuscular Disorders 19 (2009) 270-274.

22. Verburg, E., Murphy, R.M., Richard, I. and Lamb, G.D.: “Involvement of calpains in Ca2+-induced disruption of excitation-contraction coupling in mammalian skeletal muscle fibers”. Am J Physiol Cell Physiol 296 (2009) C1115-C1122.

23Veron, P., Boutin, S., Martin, S., Chaperot, L., Plumas, J., Davoust, J. and Masurier, C.:“Highly efficient transduction of human plasmacytoid dendritic cells without phenotypic and functional maturation”. J Transl Med 7 (2009) 10.

24Zanta-Boussif, M.A., Charrier, S., Brice-Ouzet, A., Martin, S., Opolon, P., Thrasher, A.J., Hope, T.J. and Galy, A.: “Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS”. Gene Ther 16 (2009) 605–619.