Journal articles on the topic 'Microdystrophin'
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Cernisova, Viktorija, Ngoc Lu-Nguyen, Jessica Trundle, Shan Herath, Alberto Malerba, and Linda Popplewell. "Microdystrophin Gene Addition Significantly Improves Muscle Functionality and Diaphragm Muscle Histopathology in a Fibrotic Mouse Model of Duchenne Muscular Dystrophy." International Journal of Molecular Sciences 24, no. 9 (May 3, 2023): 8174. http://dx.doi.org/10.3390/ijms24098174.
Full textBrown, K., M. Lawlor, D. Golebiowski, P. Gonzalez, V. Ricotti, J. Schneider, and C. Morris. "Quantification of microdystrophin and correlation to circulating biomarkers." Neuromuscular Disorders 27 (October 2017): S214. http://dx.doi.org/10.1016/j.nmd.2017.06.431.
Full textHersh, Jessica, José Manuel Condor Capcha, Camila Iansen Irion, Guerline Lambert, Mauricio Noguera, Mohit Singh, Avinash Kaur, et al. "Peptide-Functionalized Dendrimer Nanocarriers for Targeted Microdystrophin Gene Delivery." Pharmaceutics 13, no. 12 (December 15, 2021): 2159. http://dx.doi.org/10.3390/pharmaceutics13122159.
Full textHo, Peggy P., Lauren J. Lahey, Foteini Mourkioti, Peggy E. Kraft, Antonio Filareto, Moritz Brandt, Klas E. G. Magnusson, et al. "Engineered DNA plasmid reduces immunity to dystrophin while improving muscle force in a model of gene therapy of Duchenne dystrophy." Proceedings of the National Academy of Sciences 115, no. 39 (September 4, 2018): E9182—E9191. http://dx.doi.org/10.1073/pnas.1808648115.
Full textMartin, Paul T., Rui Xu, Louise R. Rodino-Klapac, Elaine Oglesbay, Marybeth Camboni, Chrystal L. Montgomery, Kim Shontz, et al. "Overexpression of Galgt2 in skeletal muscle prevents injury resulting from eccentric contractions in both mdx and wild-type mice." American Journal of Physiology-Cell Physiology 296, no. 3 (March 2009): C476—C488. http://dx.doi.org/10.1152/ajpcell.00456.2008.
Full textBostick, Brian, Jin-Hong Shin, Yongping Yue, and Dongsheng Duan. "AAV-microdystrophin Therapy Improves Cardiac Performance in Aged Female mdx Mice." Molecular Therapy 19, no. 10 (October 2011): 1826–32. http://dx.doi.org/10.1038/mt.2011.154.
Full textPercival, Justin M., Paul Gregorevic, Guy L. Odom, Glen B. Banks, Jeffrey S. Chamberlain, and Stanley C. Froehner. "rAAV6-Microdystrophin Rescues Aberrant Golgi Complex Organization in mdx Skeletal Muscles." Traffic 8, no. 10 (July 12, 2007): 1424–39. http://dx.doi.org/10.1111/j.1600-0854.2007.00622.x.
Full textBoehler, Jessica F., Valeria Ricotti, J. Patrick Gonzalez, Meghan Soustek-Kramer, Lauren Such, Kristy J. Brown, Joel S. Schneider, and Carl A. Morris. "Membrane recruitment of nNOSµ in microdystrophin gene transfer to enhance durability." Neuromuscular Disorders 29, no. 10 (October 2019): 735–41. http://dx.doi.org/10.1016/j.nmd.2019.08.009.
Full textShin, Jin-Hong, Xiufang Pan, Chady H. Hakim, Hsiao T. Yang, Yongping Yue, Keqing Zhang, Ronald L. Terjung, and Dongsheng Duan. "Microdystrophin Ameliorates Muscular Dystrophy in the Canine Model of Duchenne Muscular Dystrophy." Molecular Therapy 21, no. 4 (April 2013): 750–57. http://dx.doi.org/10.1038/mt.2012.283.
Full textPichavant, Christophe, Pierre Chapdelaine, Daniel G. Cerri, Jean-Christophe Dominique, Simon P. Quenneville, Daniel Skuk, Joe N. Kornegay, João CS Bizario, Xiao Xiao, and Jacques P. Tremblay. "Expression of Dog Microdystrophin in Mouse and Dog Muscles by Gene Therapy." Molecular Therapy 18, no. 5 (May 2010): 1002–9. http://dx.doi.org/10.1038/mt.2010.23.
Full textGregorevic, Paul, James M. Allen, Elina Minami, Michael J. Blankinship, Miki Haraguchi, Leonard Meuse, Eric Finn, et al. "rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice." Nature Medicine 12, no. 7 (July 2006): 787–89. http://dx.doi.org/10.1038/nm1439.
Full textDanilov, Kirill A., Svetlana G. Vassilieva, Anna V. Polikarpova, Anna V. Starikova, Anna A. Shmidt, Ivan I. Galkin, Alexandra A. Tsitrina, Tatiana V. Egorova, Sergei N. Orlov, and Yuri V. Kotelevtsev. "In vitro assay for the efficacy assessment of AAV vectors expressing microdystrophin." Experimental Cell Research 392, no. 2 (July 2020): 112033. http://dx.doi.org/10.1016/j.yexcr.2020.112033.
Full textShin, J.-H., Y. Nitahara-Kasahara, H. Hayashita-Kinoh, S. Ohshima-Hosoyama, K. Kinoshita, T. Chiyo, H. Okada, T. Okada, and S. Takeda. "Improvement of cardiac fibrosis in dystrophic mice by rAAV9-mediated microdystrophin transduction." Gene Therapy 18, no. 9 (March 31, 2011): 910–19. http://dx.doi.org/10.1038/gt.2011.36.
Full textBenabdallah, Basma F., Arnaud Duval, Joel Rousseau, Pierre Chapdelaine, Michael C. Holmes, Eli Haddad, Jacques P. Tremblay, and Christian M. Beauséjour. "Targeted Gene Addition of Microdystrophin in Mice Skeletal Muscle via Human Myoblast Transplantation." Molecular Therapy - Nucleic Acids 2 (2013): e68. http://dx.doi.org/10.1038/mtna.2012.55.
Full textXiong, Fu, Shaobo Xiao, Meijuan Yu, Wanyi Li, Hui Zheng, Yanchang Shang, Funing Peng, et al. "Enhanced effect of microdystrophin gene transfection by HSV-VP22 mediated intercellular protein transport." BMC Neuroscience 8, no. 1 (2007): 50. http://dx.doi.org/10.1186/1471-2202-8-50.
Full textXiong, F., Y. Xu, H. Zheng, X. Lu, S. Feng, Y. Shang, Y. Li, Y. Zhang, S. Jin, and C. Zhang. "Microdystrophin Delivery in Dystrophin-Deficient (mdx) Mice by Genetically-Corrected Syngeneic MSCs Transplantation." Transplantation Proceedings 42, no. 7 (September 2010): 2731–39. http://dx.doi.org/10.1016/j.transproceed.2010.04.031.
Full textMurray, Jason, Guy Odom, Sigurast Olafsson, Stephen Hauschka, Jeffrey Chamberlain, Farid Moussavi-Harami, and Michael Regnier. "AAV-Mediated Delivery of Ribonucleotide Reductase and Microdystrophin Rescues Function in Dystrophic Mice." Biophysical Journal 114, no. 3 (February 2018): 541a. http://dx.doi.org/10.1016/j.bpj.2017.11.2956.
Full textKoo, Taeyoung, Takashi Okada, Takis Athanasopoulos, Helen Foster, Shin'ichi Takeda, and George Dickson. "Long-term functional adeno-associated virus-microdystrophin expression in the dystrophic CXMDj dog." Journal of Gene Medicine 13, no. 9 (September 2011): 497–506. http://dx.doi.org/10.1002/jgm.1602.
Full textWilton-Clark, Harry, and Toshifumi Yokota. "Antisense and Gene Therapy Options for Duchenne Muscular Dystrophy Arising from Mutations in the N-Terminal Hotspot." Genes 13, no. 2 (January 28, 2022): 257. http://dx.doi.org/10.3390/genes13020257.
Full textHamm, Shelby E., Daniel D. Fathalikhani, Katherine E. Bukovec, Adele K. Addington, Haiyan Zhang, Justin B. Perry, Ryan P. McMillan, et al. "Voluntary wheel running complements microdystrophin gene therapy to improve muscle function in mdx mice." Molecular Therapy - Methods & Clinical Development 23 (December 2021): 460. http://dx.doi.org/10.1016/j.omtm.2021.10.005.
Full textHamm, Shelby E., Daniel D. Fathalikhani, Katherine E. Bukovec, Adele K. Addington, Haiyan Zhang, Justin B. Perry, Ryan P. McMillan, et al. "Voluntary wheel running complements microdystrophin gene therapy to improve muscle function in mdx mice." Molecular Therapy - Methods & Clinical Development 21 (June 2021): 144–60. http://dx.doi.org/10.1016/j.omtm.2021.02.024.
Full textBostick, Brian, Yongping Yue, Yi Lai, Chun Long, Dejia Li, and Dongsheng Duan. "Adeno-Associated Virus Serotype-9 Microdystrophin Gene Therapy Ameliorates Electrocardiographic Abnormalities in mdx Mice." Human Gene Therapy 19, no. 8 (August 2008): 851–56. http://dx.doi.org/10.1089/hum.2008.058.
Full textLiu, Mingju, Yongping Yue, Scott Q. Harper, Robert W. Grange, Jeffrey S. Chamberlain, and Dongsheng Duan. "Adeno-Associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury." Molecular Therapy 11, no. 2 (February 2005): 245–56. http://dx.doi.org/10.1016/j.ymthe.2004.09.013.
Full textAbmayr, Simone, Paul Gregorevic, James M. Allen, and Jeffrey S. Chamberlain. "Phenotypic Improvement of Dystrophic Muscles by rAAV/Microdystrophin Vectors Is Augmented by Igf1 Codelivery." Molecular Therapy 12, no. 3 (September 2005): 441–50. http://dx.doi.org/10.1016/j.ymthe.2005.04.001.
Full textBachrach, E., S. Li, A. L. Perez, J. Schienda, K. Liadaki, J. Volinski, A. Flint, J. Chamberlain, and L. M. Kunkel. "Systemic delivery of human microdystrophin to regenerating mouse dystrophic muscle by muscle progenitor cells." Proceedings of the National Academy of Sciences 101, no. 10 (March 1, 2004): 3581–86. http://dx.doi.org/10.1073/pnas.0400373101.
Full textWillcocks, R., D. Lott, S. Forbes, K. Vandenborne, and G. Walter. "399P MRI assessment of microdystrophin gene therapy in DMD: a five year longitudinal study." Neuromuscular Disorders 43 (October 2024): 104441.127. http://dx.doi.org/10.1016/j.nmd.2024.07.136.
Full textChicoine, LG, CL Montgomery, WG Bremer, KM Shontz, DA Griffin, KN Heller, S. Lewis, et al. "Plasmapheresis Eliminates the Negative Impact of AAV Antibodies on Microdystrophin Gene Expression Following Vascular Delivery." Molecular Therapy 22, no. 2 (February 2014): 338–47. http://dx.doi.org/10.1038/mt.2013.244.
Full textGregorevic, Paul, Michael J. Blankinship, James M. Allen, and Jeffrey S. Chamberlain. "Systemic Microdystrophin Gene Delivery Improves Skeletal Muscle Structure and Function in Old Dystrophic mdx Mice." Molecular Therapy 16, no. 4 (April 2008): 657–64. http://dx.doi.org/10.1038/mt.2008.28.
Full textJørgensen, Louise H., Nancy Larochelle, Kristian Orlopp, Patrick Dunant, Roy W. R. Dudley, Rolf Stucka, Christian Thirion, Maggie C. Walter, Steven H. Laval, and Hanns Lochmüller. "Efficient and Fast Functional Screening of Microdystrophin ConstructsIn VivoandIn Vitrofor Therapy of Duchenne Muscular Dystrophy." Human Gene Therapy 20, no. 6 (June 2009): 641–50. http://dx.doi.org/10.1089/hum.2008.162.
Full textXIONG, F., C. ZHANG, S. XIAO, M. LI, S. WANG, M. YU, and Y. SHANG. "Construction of Recombinant Adenovirus Including Microdystrophin and Expression in the Mesenchymal Cells of mdx Mice." Chinese Journal of Biotechnology 23, no. 1 (January 2007): 27–32. http://dx.doi.org/10.1016/s1872-2075(07)60003-x.
Full textHayashita-Kinoh, Hiromi, Posadas-Herrera Guillermo, Yuko Nitahara-Kasahara, Mutsuki Kuraoka, Hironori Okada, Tomoko Chiyo, Shin’ichi Takeda, and Takashi Okada. "Improved transduction of canine X-linked muscular dystrophy with rAAV9-microdystrophin via multipotent MSC pretreatment." Molecular Therapy - Methods & Clinical Development 20 (March 2021): 133–41. http://dx.doi.org/10.1016/j.omtm.2020.11.003.
Full textFeng, Shan-wei, Fei Chen, Jiqing Cao, Mei-juan Yu, Ying-yin Liang, Xin-ming Song, and Cheng Zhang. "Restoration of muscle fibers and satellite cells after isogenic MSC transplantation with microdystrophin gene delivery." Biochemical and Biophysical Research Communications 419, no. 1 (March 2012): 1–6. http://dx.doi.org/10.1016/j.bbrc.2012.01.029.
Full textHayashita-Kinoh, Hiromi, Hironori Okada, Yuko N. Kasahara, Tomoko Chiyo, Kiwamu Imagawa, Katsuhiko Tachibana, Shin'ichi Takeda, and Takashi Okada. "378. Improved Transduction of Canine X-Linked Muscular Dystrophy with rAAV9-Microdystrophin by Introducing Immune Tolerance." Molecular Therapy 24 (May 2016): S150—S151. http://dx.doi.org/10.1016/s1525-0016(16)33187-2.
Full textHayashita-Kinoh, Hiromi, Hironori Okada, Yuko Nitahara-Kasahara, Tomoko Chiyo, Kiwamu Imagawa, Katsuhiko Tachibana, Shin'ichi Takeda, and Takashi Okada. "400. Improved Transduction of Canine X-Linked Muscular Dystrophy With rAAV9-Microdystrophin By Using MSCs Pretreatment." Molecular Therapy 23 (May 2015): S158—S159. http://dx.doi.org/10.1016/s1525-0016(16)34009-6.
Full textYoshimura, Madoka, Miki Sakamoto, Madoka Ikemoto, Yasushi Mochizuki, Katsutoshi Yuasa, Yuko Miyagoe-Suzuki, and Shin'ichi Takeda. "AAV vector-mediated microdystrophin expression in a relatively small percentage of mdx myofibers improved the mdx phenotype." Molecular Therapy 10, no. 5 (November 2004): 821–28. http://dx.doi.org/10.1016/j.ymthe.2004.07.025.
Full textYue, Yongping, Zhenbo Li, Scott Q. Harper, Robin L. Davisson, Jeffrey S. Chamberlain, and Dongsheng Duan. "Microdystrophin Gene Therapy of Cardiomyopathy Restores Dystrophin-Glycoprotein Complex and Improves Sarcolemma Integrity in the Mdx Mouse Heart." Circulation 108, no. 13 (September 30, 2003): 1626–32. http://dx.doi.org/10.1161/01.cir.0000089371.11664.27.
Full textXiong, Fu, Shaobo Xiao, Funing Peng, Hui Zheng, Meijuan Yu, Yechun Ruan, Wanyi Li, et al. "Herpes Simplex Virus VP22 Enhances Adenovirus-Mediated Microdystrophin Gene Transfer to Skeletal Muscles in Dystrophin-Deficient (mdx) Mice." Human Gene Therapy 18, no. 6 (June 2007): 490–501. http://dx.doi.org/10.1089/hum.2006.155.
Full textHayashita-Kinoh, Hiromi, Naoko Yugeta, Hironori Okada, Yuko Nitahara-Kasahara, Tomoko Chiyo, Takashi Okada, and Shin'ichi Takeda. "Intra-Amniotic rAAV-Mediated Microdystrophin Gene Transfer Improves Canine X-Linked Muscular Dystrophy and May Induce Immune Tolerance." Molecular Therapy 23, no. 4 (April 2015): 627–37. http://dx.doi.org/10.1038/mt.2015.5.
Full textDastgir, J., S. Rastogi, D. Philips, C. Wilson, N. Boulos, J. Hall, V. Jimenez, et al. "P16 An investigational AAV8 gene therapy coding for a novel microdystrophin as a treatment for Duchenne muscular dystrophy." Neuromuscular Disorders 33 (October 2023): S101. http://dx.doi.org/10.1016/j.nmd.2023.07.143.
Full textFilareto, Antonio, Katie Maguire-Nguyen, Qiang Gan, Garazi Aldanondo, Léo Machado, Jeffrey S. Chamberlain, and Thomas A. Rando. "Monitoring disease activity noninvasively in the mdx model of Duchenne muscular dystrophy." Proceedings of the National Academy of Sciences 115, no. 30 (July 9, 2018): 7741–46. http://dx.doi.org/10.1073/pnas.1802425115.
Full textSchinkel, Stefanie, Ralf Bauer, Raffi Bekeredjian, Rolf Stucka, Désirée Rutschow, Hanns Lochmüller, Jürgen A. Kleinschmidt, Hugo A. Katus, and Oliver J. Müller. "Long-Term Preservation of Cardiac Structure and Function After Adeno-Associated Virus Serotype 9-Mediated Microdystrophin Gene Transfer inmdxMice." Human Gene Therapy 23, no. 6 (June 2012): 566–75. http://dx.doi.org/10.1089/hum.2011.017.
Full textAthanasopoulos, Takis, Ian Graham, Capucine Trollet, Helen Foster, Norma Perez, Vanessa Hill, Phillippe Moullier, and George Dickson. "907. Development of Recombinant Novel Adeno-Associated Viral (rAAV) Vectors Encoding Optimised Microdystrophin cDNAs for Duchenne Muscular Dystrophy (DMD)." Molecular Therapy 13 (2006): S349—S350. http://dx.doi.org/10.1016/j.ymthe.2006.08.997.
Full textLe Guiner, C., M. Montus, L. Servais, Y. Cherel, J. Y. Hogrel, P. Carlier, C. Masurier, et al. "P.20.13 Gene therapy of Duchenne Muscular Dystrophy using rAAV vectors: Exon skipping and microdystrophin approaches in GRMD dogs." Neuromuscular Disorders 23, no. 9-10 (October 2013): 842–43. http://dx.doi.org/10.1016/j.nmd.2013.06.703.
Full textBourg, Nathalie, Ai Vu Hong, William Lostal, Abbass Jaber, Nicolas Guerchet, Guillaume Tanniou, Fanny Bordier, et al. "Co-Administration of Simvastatin Does Not Potentiate the Benefit of Gene Therapy in the mdx Mouse Model for Duchenne Muscular Dystrophy." International Journal of Molecular Sciences 23, no. 4 (February 11, 2022): 2016. http://dx.doi.org/10.3390/ijms23042016.
Full textDastgir, J., P. Falabella, C. Qiao, S. Kim, N. Buss, M. Fiscella, S. Pakola, and O. Danos. "P.130 RGX-202: An investigational AAV8 gene therapy coding for a novel microdystrophin as a treatment for Duchenne muscular dystrophy." Neuromuscular Disorders 32 (October 2022): S101. http://dx.doi.org/10.1016/j.nmd.2022.07.246.
Full textDreghici, R. Donisa, S. Redican, J. Lawrence, K. Brown, F. Wang, J. Gonzalez, J. Schneider, C. Morris, P. Shieh, and B. Byrne. "FP.28 IGNITE DMD phase I/II study of SGT-001 microdystrophin gene therapy for DMD: Long-term outcomes and expression update." Neuromuscular Disorders 32 (October 2022): S98. http://dx.doi.org/10.1016/j.nmd.2022.07.234.
Full textFoster, Helen, Paul S. Sharp, Takis Athanasopoulos, Capucine Trollet, Ian R. Graham, Keith Foster, Dominic J. Wells, and George Dickson. "Codon and mRNA Sequence Optimization of Microdystrophin Transgenes Improves Expression and Physiological Outcome in Dystrophic mdx Mice Following AAV2/8 Gene Transfer." Molecular Therapy 16, no. 11 (November 2008): 1825–32. http://dx.doi.org/10.1038/mt.2008.186.
Full textGregorevic, Paul, Michael J. Blankinship, Elina Minami, James M. Allen, Charles E. Murry, and Jeffrey S. Chamberlain. "35. Systemic Administration of rAAV6-Microdystrophin Preserves Muscle Function and Extends Lifespan in the Dystrophin-/Utrophin- Mouse Model of Severe Muscular Dystrophy." Molecular Therapy 13 (2006): S15. http://dx.doi.org/10.1016/j.ymthe.2006.08.048.
Full textFoster, H., D. J. Wells, C. Trollet, T. Athanasopoulos, I. Graham, K. Foster, and J. G. Dickson. "G.P.8.08 Codon optimisation of microdystrophin results in improvements in expression and physiological outcome in the mdx mouse following AAV8 gene transfer." Neuromuscular Disorders 18, no. 9-10 (October 2008): 784. http://dx.doi.org/10.1016/j.nmd.2008.06.207.
Full textLe Guiner, C., L. Servais, M. Montus, F. Bodvael, B. Gjata, J. Y. Hogrel, P. Carlier, et al. "Adeno-associated virus vector (AAV) microdystrophin gene therapy prolongs survival and restores muscle function in the canine model of Duchenne muscular dystrophy (DMD)." Neuromuscular Disorders 25 (October 2015): S315. http://dx.doi.org/10.1016/j.nmd.2015.06.458.
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