Scientific Publications 2010

1. Beggs, A.H., J. Bohm, E. Snead, M. Kozlowski, M. Maurer, K. Minor, M.K. Childers, S.M. Taylor, C. Hitte, J.R. Mickelson, L.T. Guo, A.P. Mizisin, A. Buj-Bello, L. Tiret, J. Laporte, and G.D. Shelton,“MTM1 mutation associated with X-linked myotubular myopathy in Labrador Retrievers”. Proc Natl Acad Sci U S A, 2010. 107(33): p. 14697-14702.

2Benkhelifa-Ziyyat, S., S. Bucher, M.A. Zanta-Boussif, J. Pasquet, and O. Danos, “Changes in the accessibility of the HIV-1 Integrase C-terminus in the presence of cellular proteins”. Retrovirology, 2010. 7(1): p. 27.

3. Bolduc, V., G. Marlow, K.M. Boycott, K. Saleki, H. Inoue, J. Kroon, M. Itakura, Y. Robitaille, L. Parent, F. Baas, K. Mizuta, N. Kamata, I. Richard, W.H. Linssen, I. Mahjneh, M. de Visser, R. Bashir, and B. Brais, “Recessive Mutations in the Putative Calcium-Activated Chloride Channel Anoctamin 5 Cause Proximal LGMD2L and Distal MMD3 Muscular Dystrophies”. Am J Hum Genet, 2010. 86(2): p. 213-221.

4Boutin, S., V. Monteilhet, P. Veron, C. Leborgne, O. Benveniste, M.F. Montus, and C. Masurier“Prevalence of serum IgG and neutralizing factors against adeno-associated virus types 1, 2, 5, 6, 8 and 9 in the healthy population: implications for gene therapy using AAV vectors”. Hum Gene Ther, 2010. 21(6): p. 704-712.

5Charton, K., N. Daniele, A. Vihola, C. Roudaut, E. Gicquel, F. Monjaret, A. Tarrade, J. Sarparanta, B. Udd, and I. Richard“Removal of the calpain 3 protease reverses the myopathology in a mouse model for titinopathies”. Hum Mol Genet, 2010. 19(23): p. 4608-4624.

6. Capron, C., C. Lacout, Y. Lecluse, V. Jalbert, H. Chagraoui, S. Charrier, A. Galy, A. Bennaceur-Griscelli, E. Cramer-Borde, and W. Vainchenker, “A major role of TGF-{beta}1 in the homing capacities of murine hematopoietic stem cell/ progenitors”. Blood, 2010. 116(8): p. 1244-1253.

7. Claeys, K.G., M. Sozanska, J.J. Martin, E. Lacene, L. Vignaud, D. Stockholm, P. Laforet, B. Eymard, A. Kichler, D. Scherman, T. Voit, and D. Israeli, “DNAJB2 Expression in Normal and Diseased Human and Mouse Skeletal Muscle”. Am J Pathol, 2010. 176(6): p. 2901-2910.

8.  Hauser, H., M. Carrondo, D. Wirth, A.S. Coroadinha, P. Cruz, H. Falk, R. Schucht, F. Dupont, C. Geny-Fiamma, and O.W. Merten“Integrated strategy for the production of therapeutic retroviral vectors”. Hum Gene Ther, 2010. Online Ahead of Editing: November 2.

9. Klein, E., M. Ciobanu, J. Klein, V. Machi, C. Leborgne, T. Vandamme, B. Frisch, F. Pons, A. Kichler, G. Zuber, and L. Lebeau, “”HFP” Fluorinated Cationic Lipids for Enhanced Lipoplex Stability and Gene Delivery”. Bioconjug Chem, 2010. 21(2): p. 360-71.

10. Klein, E., C. Leborgne, M. Ciobanu, J. Klein, B. Frisch, F. Pons, G. Zuber, D. Scherman, A. Kichler, and L. Lebeau, “Nucleic acid transfer with hemifluorinated polycationic lipids”. Biomaterials, 2010. 31(17): p. 4781-8.

11. Krahn, M., N. Wein, M. BartoliW. Lostal, S. Courrier, N. Bourg-Alibert, K. Nguyen, C. Vial, N. Streichenberger, V. Labelle, D. DePetris, C. Pécheux, F. Leturcq, P. Cau, I. Richard, and N. Lévy,“A Naturally Occurring Human Minidysferlin Protein Repairs Sarcolemmal Lesions in a Mouse Model of Dysferlinopathy”. Science Translational Medicine, 2010. 2(50): p. 50ra69.

12. Lan, Y., B. Langlet-Bertin, V. Abbate, L.S. Vermeer, X. Kong, K.E. Sullivan, C. LeborgneD. Scherman, R.C. Hider, A.F. Drake, S.S. Bansal, A. Kichler, and A.J. Mason, “Incorporation of 2,3-Diaminopropionic Acid into Linear Cationic Amphipathic Peptides Produces pH-Sensitive Vectors”. Chembiochem, 2010. 11(9): p. 1266 – 1272.

13. Langlet-Bertin, B., C. Leborgne, D. Scherman, B. Bechinger, A.J. Mason, and A. Kichler,“Design and Evaluation of Histidine-Rich Amphipathic Peptides for siRNA Delivery”. Pharm Res, 2010. 27(7): p. 1426-1436.

14Laure, L., N. Daniele, L. Suel, S. Marchand, S. Aubert, N. Bourg, C. Roudaut, S. Duguez, M. Bartoli, and I. Richard“A new pathway encompassing calpain 3 and its newly identified substrate cardiac ankyrin repeat protein is involved in the regulation of the nuclear factor-kappaB pathway in skeletal muscle”. FEBS J, 2010. 277(20): p. 4322-4337.

15. Lidgi-Guigui, N., C. Guis, B. Brissault, A. Kichler, C. Leborgne, D. Scherman, S. Labdi, and P.A. Curmi, “Investigation of DNA Condensing Properties of Amphiphilic Triblock Cationic Polymers by Atomic Force Microscopy”. Langmuir, 2010. 26(22): p. 17552-17557.

16. Lock, M., S. McGorray, A. Auricchio, E. Ayuso, E.J. Beecham, V. Blouin-Tavel, F. Bosch, M. Bose, B.J. Byrne, T. Caton, J.A. Chiorini, A. Chtarto, K.R. Clark, T. Conlon, C. Darmon, M. Doria, A. Douar, T.R. Flotte, J.D. Francis, A. Francois, M. Giacca, M.T. Korn, I. Korytov, X. Leon, B. Leuchs, G. Lux, C. Melas, H. Mizukami, P. Moullier, M. Müller, K. Ozawa, T. Philipsberg, K. Poulard, C. Raupp, C. Rivière, S.D. Roosendaal, R.J. Samulski, S.M. Soltys, R. Surosky, L. Tenenbaum, D.L. Thomas, B. van Montfort, G. Veres, J.F. Wright, Y. Xu, O. Zelenaia, L. Zentilin, and R.O. Snyder,“Characterization of a Recombinant Adeno-Associated Virus Type 2 Reference Standard Material”. Human Gene Therapy, 2010. 21(10): p. 1273-1285.

17Lostal, W., M. Bartoli, N. Bourg, C. Roudaut, A. Bentaib, K. Miyake, N. Guerchet, F. Fougerousse, P. McNeil, and I. Richard, “Efficient recovery of dysferlin deficiency by dual adeno-associated vectors mediated gene transfer”. Hum. Mol. Genet., 2010. 19(10): p. 1897-1907.

18Moulay, G., C. Masurier, P. Bigey, D. Scherman, and A. Kichler, “Soluble TNF-[alpha] receptor secretion from healthy or dystrophic mice after AAV6-mediated muscle gene transfer”. Gene Ther, 2010. 17(11): p. 1400–1410.

19. Sarparanta, J., G. Blandin, K. Charton, A. Vihola, S. Marchand, A. Milic, P. Hackman, E. Ehler, I. Richard, and B. Udd, “Interactions with M-band titin and calpain 3 link myospryn (CMYA5) to tibial and limb-girdle muscular dystrophies”. J Biol Chem, 2010. 285(39): p. 30304-30315.

20. Schweizer, M. and O.W. Merten, “Large-Scale Production Means for the Manufacturing of Lentiviral Vectors”. Curr Gene Ther, 2010. 10(6): p. 474-486.

21. Wasungu, L., A.L. Marty, M.F. Bureau, A. Kichler, M. Bessodes, J. Teissie, D. Scherman, M.P. Rols, and N. Mignet, “Pre-treatment of cells with pluronic L64 increases DNA transfection mediated by electrotransfer”. J Control Release, 2010. 149(2): p. 117-125.