Chemical Chemical characteristics Advantages Disadvantages Negatively charged Good solubility – long half-life Nuclease and RNase H resistant Do not enter the heart Toxicity Uncharged Relatively safe Nuclease and RNase H…
Fig. 3.1 Multisystemic symptoms of DM 3.2 Clinical Features 3.2.1 Adult-Onset DM1 3.2.1.1 Muscle Weakness The predominant symptom of adult-onset DM1 is facial and distal muscle weakness. Facial weakness is…
Fig. 12.1 Main purposes and resources of the MDCTN As of April 2015, 33 clinical institutes distributed over all four main islands of Japan have joined the MDCTN (Fig. 12.2); these…
Fig. 10.1 Annual changes in motor function. Right column, ambulant patients; left column, nonambulant patients. a 6MWT; b, c summed MMT; d, e grip power; and f, g %FVC. All…
Fig. 8.1 Myoblast transplantation therapy (MMT) for DMD. Endogenous skeletal muscle satellite cells are located between the basal lamina and sarcolemma of myofibers in a dormant state. Upon muscle injury,…
Fig. 4.1 RV in GNE myopathy muscle. Bar 20 μm Fig. 4.2 Electron microscopic findings of GNE myopathy muscle. Autophagic vacuoles (black arrow head) are seen in the regions with various…
Fig. 2.1 Schematic representation of the dystrophin-glycoprotein complex and dystroglycan Mutations in components of the DGC, such as dystrophin and sarcoglycans, were identified to be associated with Duchenne/Becker and limb-girdle…
© Springer Japan 2016Shin’ichi Takeda, Yuko Miyagoe-Suzuki and Madoka Mori-Yoshimura (eds.)Translational Research in Muscular Dystrophy10.1007/978-4-431-55678-7_9 9. Therapeutic Approach of iPS Cell Technology for Treating Muscular Dystrophy Hidetoshi Sakurai1 (1) Department of Clinical Application, Center for iPS Cell…
Fig. 6.1 Mechanism of exon skipping therapy in DMD and DYSF. The deletion of exon 52 in the DMD gene results in an out-of-frame transcript that does not produce functional…
Fig. 1.1 Facial appearance changes with age in a girl with FCMD; images were obtained at 7 months (A), at 1 year and 5 months (B), and at 8 years…
