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AAV-directed muscular dystrophy gene therapy

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Pages 395-408 | Published online: 04 Feb 2010

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Guoxi Zheng, Zhu Zhu, Kang Zhu, Junrong Wei, Yang Jing & Maoli Duan. (2013) Therapeutic effect of adeno-associated virus (AAV)-mediated ADNF-9 expression on cochlea of kanamycin-deafened guinea pigs. Acta Oto-Laryngologica 133:10, pages 1022-1029.
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Theresa M. Raimondo & David J. Mooney. (2021) Anti-inflammatory nanoparticles significantly improve muscle function in a murine model of advanced muscular dystrophy. Science Advances 7:26.
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Xi Yin, Ying Tang, Jian Li, Anna T. Dzuricky, Chuanqiang Pu, Freddie Fu & Bing Wang. (2017) Genetic ablation of P65 subunit of NF‐κB in mdx mice to improve muscle physiological function . Muscle & Nerve 56:4, pages 759-767.
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François Monjaret, Nathalie Bourg, Laurence Suel, Carinne Roudaut, Florence Le Roy, Isabelle Richard & Karine Charton. (2014) Cis -splicing and Translation of the Pre- Trans -splicing Molecule Combine With Efficiency in Spliceosome-mediated RNA Trans -splicing. Molecular Therapy 22:6, pages 1176-1187.
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Wenjing Zhang, Yang Wang, Shuyun Dong, Rajarshi Choudhury, Yongfeng Jin & Zefeng Wang. (2014) Treatment of Type 1 Myotonic Dystrophy by Engineering Site-specific RNA Endonucleases that Target (CUG) n Repeats. Molecular Therapy 22:2, pages 312-320.
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Sandra L. HarperSira SriswasdiHsin-Yao TangMassimiliano Gaetani, Patrick G. Gallagher & David W. Speicher. (2013) The common hereditary elliptocytosis-associated α-spectrin L260P mutation perturbs erythrocyte membranes by stabilizing spectrin in the closed dimer conformation. Blood 122:17, pages 3045-3053.
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J. J. Magaña, L. Velázquez-Pérez & B. Cisneros. (2012) Spinocerebellar Ataxia Type 2: Clinical Presentation, Molecular Mechanisms, and Therapeutic Perspectives. Molecular Neurobiology 47:1, pages 90-104.
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Christine Rivat, Adrian J. Thrasher & H. Bobby Gaspar. 2013. Clinical Immunology. Clinical Immunology 1054 1065 .
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Ingrid E.C. Verhaart & Annemieke Aartsma-Rus. (2012) Gene therapy for Duchenne muscular dystrophy. Current Opinion in Neurology 25:5, pages 588-596.
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Bilal A. Azakir, Sabrina Di Fulvio, Steven Salomon, Marielle Brockhoff, Christian Therrien & Michael Sinnreich. (2012) Modular Dispensability of Dysferlin C2 Domains Reveals Rational Design for Mini-dysferlin Molecules. Journal of Biological Chemistry 287:33, pages 27629-27636.
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Reiko Arakawa, Ryoko Aoki, Masayuki Arakawa & Kayoko Saito. (2012) Human first-trimester chorionic villi have a myogenic potential. Cell and Tissue Research 348:1, pages 189-197.
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Anna L. David & Simon N. Waddington. 2012. Prenatal Gene Therapy. Prenatal Gene Therapy 9 39 .
Dwi U Kemaladewi, Willem MH Hoogaars, Sandra H van Heiningen, Samuel Terlouw, David JJ de Gorter, Johan T den Dunnen, Gert Jan B van Ommen, Annemieke Aartsma-Rus, Peter ten Dijke & Peter AC 't Hoen. (2011) Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy. BMC Medical Genomics 4:1.
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Farhang Alaee, Osamu Sugiyama, Mandeep S Virk, Ying Tang, Bing Wang & Jay R Lieberman. (2011) In vitro evaluation of a double-stranded self-complementary adeno-associated virus type2 vector in bone marrow stromal cells for bone healing. Genetic Vaccines and Therapy 9:1.
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Bo Wu, Bin Xiao, Caryn Cloer, Mona Shaban, Arpana Sali, Peijuan Lu, Juan Li, Kanneboyina Nagaraju, Xiao Xiao & Qi Long Lu. (2011) One-year Treatment of Morpholino Antisense Oligomer Improves Skeletal and Cardiac Muscle Functions in Dystrophic mdx Mice. Molecular Therapy 19:3, pages 576-583.
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Jonathan J. Magaña & Bulmaro Cisneros. (2011) Perspectives on gene therapy in myotonic dystrophy type 1. Journal of Neuroscience Research 89:3, pages 275-285.
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Markus A. RüeggDavid J. Glass. (2011) Molecular Mechanisms and Treatment Options for Muscle Wasting Diseases. Annual Review of Pharmacology and Toxicology 51:1, pages 373-395.
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