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Dmd ips

Nadine Matthias, Samuel D Hunt, Jianbo Wu, Radbod Darabi
Muscular dystrophies are among major inherited muscle disorders characterized by progressive muscle damage and fibrosis with no definitive cure. Recently, gene or cell based therapies have been developed to restore the missing gene expression or replace the damaged tissues. In order to test the efficiency of these therapies in mice models of muscular dystrophies, the arterial route of delivery is very advantageous as it provides uniform muscle exposure to the therapeutic agents or cells. Although there are few reports of arterial delivery of the therapeutic agents or cells in mice, there is no in-depth description and evaluation of its efficacy in perfusion of downstream muscles...
November 15, 2015: Experimental Cell Research
Bo Lin, Yang Li, Lu Han, Aaron D Kaplan, Ying Ao, Spandan Kalra, Glenna C L Bett, Randall L Rasmusson, Chris Denning, Lei Yang
Duchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin gene (DMD), and is characterized by progressive weakness in skeletal and cardiac muscles. Currently, dilated cardiomyopathy due to cardiac muscle loss is one of the major causes of lethality in late-stage DMD patients. To study the molecular mechanisms underlying dilated cardiomyopathy in DMD heart, we generated cardiomyocytes (CMs) from DMD and healthy control induced pluripotent stem cells (iPSCs). DMD iPSC-derived CMs (iPSC-CMs) displayed dystrophin deficiency, as well as the elevated levels of resting Ca(2+), mitochondrial damage and cell apoptosis...
May 2015: Disease Models & Mechanisms
Yumei Luo, Yong Fan, Xinjie Chen, Lei Yue, Bolan Yu, Qing Li, Yaoyong Chen, Xiaofang Sun
The generation of disease-specific induced pluripotent stem cell (iPS cell) lines from patients with incurable diseases is a promising approach for studying disease mechanisms and for drug screening. Such innovation enables us to obtain autologous cell sources for regenerative medicine. Herein, we report the generation and characterization of iPS cells from the fibroblasts of patients with a family history of Duchenne muscular dystrophy (DMD); these fibroblasts were obtained from patients at 22 gestational weeks of age and exhibit exon duplication from exons 16 to 42...
January 2014: International Journal of Neuroscience
Mitsuo Oshimura, Yasuhiro Kazuki, Narumi Uno
Human artificial chromosomes (HACs) are stable episomal gene vectors that can carry large gene inserts. We have reported complete correction of a genetic deficiency following the transfer of a HAC carrying the genomic dystrophin sequence (DYS-HAC) into induced pluripotent stem (iPS) cells derived from either a Duchenne muscular dystrophy (DMD) model mouse or a human DMD patient. The engineered iPS cells could differentiate in immunodeficient nude mice, and human dystrophin expression was detected in muscle-like tissues...
2012: Rinshō Shinkeigaku, Clinical Neurology
Yasuhiro Kazuki, Mitsuo Oshimura
Human artificial chromosomes (HACs) exhibit several potential characteristics desired for an ideal gene delivery vector, including stable episomal maintenance and the capacity to carry large genomic loci with their regulatory elements, thus allowing the physiological regulation of the introduced gene. Stem cells, including hematopoietic stem cells, bone marrow-derived MSCs and iPS cells, can potentially avoid immune rejection due to their being obtained from the patient's own tissues. Recently, we succeeded the complete correction of a genetic deficiency in iPS cells derived from a human Duchenne muscular dystrophy patient using the HAC technology...
December 2011: Nihon Rinsho. Japanese Journal of Clinical Medicine
Takashi Nishiyama, Shin'ichi Takeda
Duchenne muscular dystrophy (DMD) is a devastating muscle disorder caused by mutations in the dystrophin gene. There is currently no effective treatment for DMD. Muscle satellite cells are tissue-specific stem cells found in the skeletal muscle; these cells play a central role in postnatal muscle growth and regeneration, and are, therefore, a potential source for stem cell therapy for DMD. However, transplantation of satellite cell-derived myoblasts has not yet been successful in humans. Patient-specific induced pluripotent stem (iPS) cells are expected to be a source for autologous cell transplantation therapy for DMD, because iPS cells can proliferate vigorously in vitro and can differentiate into multiple cell lineages both in vitro and in vivo...
January 2012: Brain and Nerve, Shinkei Kenkyū No Shinpo
Fei Chen, Jiqing Cao, Qiang Liu, Jie Qin, Jie Kong, Yanyun Wang, Yaqin Li, Jia Geng, Qiuling Li, Liqing Yang, Andy Peng Xiang, Cheng Zhang
Recently, induced pluripotent stem cells (iPS cells) have been derived from various techniques and show great potential for therapy of human diseases. Furthermore, the iPS technique can be used to provide cell models to explore pathological mechanisms of many human diseases in vitro, such as Duchenne muscular dystrophy (DMD), which is a severe recessive X-linked form of muscular dystrophy without effective treatment. In this study, we try to determine whether there are different characteristics of myocytes from mdx iPS cells and C57BL/10 iPS cells...
February 2012: Journal of Cellular Biochemistry
Tatsutoshi Nakahata, Tomonari Awaya, Hsi Chang, Yuta Mizuno, Akira Niwa, So-ichiro Fukada, Shin'ichi Takeda, Shinya Yamanaka, Toshio Heike
No abstract text is available yet for this article.
November 2010: Rinshō Shinkeigaku, Clinical Neurology
Chang Hee Lee, Lauren M Brubaker, David A Gerber, Young Mi Ku, Young Hoon Kim, Sang Soo Shin, Richard C Semelka
PURPOSE: To describe the patterns of recurrence and serial magnetic resonance imaging (MRI) features of hepatocellular carcinoma (HCC) after liver transplantation. MATERIALS AND METHODS: All cases of recurrent HCC after transplantation between September 2002 and August 2009 that underwent MRI including precontrast T1, T2-weighted images, and postgadolinium dynamic images were reviewed. On MRI we evaluated the characteristics and patterns of recurrent HCC after transplantation...
June 2011: Journal of Magnetic Resonance Imaging: JMRI
Y Even, J L Bennett, S Sekulovic, L So, L Yi, K McNagny, R K Humphries, F M V Rossi
Gene therapy as well as methods capable of returning cells to a pluripotent state (iPS) have enabled the correction of genetic deficiencies in syngenic adult progenitors, reducing the need for immunosuppression in cell therapy approaches. However, in diseases involving mutations that lead to the complete lack of a protein, such as Duchenne muscular dystrophy, the main immunogens leading to rejection of transplanted cells are the therapeutic proteins themselves. In these cases even iPS cells would not circumvent the need for immunosuppression, and alternative strategies must be developed...
2011: Cell Transplantation
In-Hyun Park
No abstract text is available yet for this article.
February 2010: Molecular Therapy: the Journal of the American Society of Gene Therapy
Yasuhiro Kazuki, Masaharu Hiratsuka, Masato Takiguchi, Mitsuhiko Osaki, Naoyo Kajitani, Hidetoshi Hoshiya, Kei Hiramatsu, Toko Yoshino, Kanako Kazuki, Chie Ishihara, Shoko Takehara, Katsumi Higaki, Masato Nakagawa, Kazutoshi Takahashi, Shinya Yamanaka, Mitsuo Oshimura
Human artificial chromosome (HAC) has several advantages as a gene therapy vector, including stable episomal maintenance that avoids insertional mutations and the ability to carry large gene inserts including the regulatory elements. Induced pluripotent stem (iPS) cells have great potential for gene therapy, as such cells can be generated from the individual's own tissues, and when reintroduced can contribute to the specialized function of any tissue. As a proof of concept, we show herein the complete correction of a genetic deficiency in iPS cells derived from Duchenne muscular dystrophy (DMD) model (mdx) mice and a human DMD patient using a HAC with a complete genomic dystrophin sequence (DYS-HAC)...
February 2010: Molecular Therapy: the Journal of the American Society of Gene Therapy
In-Hyun Park, Natasha Arora, Hongguang Huo, Nimet Maherali, Tim Ahfeldt, Akiko Shimamura, M William Lensch, Chad Cowan, Konrad Hochedlinger, George Q Daley
Tissue culture of immortal cell strains from diseased patients is an invaluable resource for medical research but is largely limited to tumor cell lines or transformed derivatives of native tissues. Here we describe the generation of induced pluripotent stem (iPS) cells from patients with a variety of genetic diseases with either Mendelian or complex inheritance; these diseases include adenosine deaminase deficiency-related severe combined immunodeficiency (ADA-SCID), Shwachman-Bodian-Diamond syndrome (SBDS), Gaucher disease (GD) type III, Duchenne (DMD) and Becker muscular dystrophy (BMD), Parkinson disease (PD), Huntington disease (HD), juvenile-onset, type 1 diabetes mellitus (JDM), Down syndrome (DS)/trisomy 21, and the carrier state of Lesch-Nyhan syndrome...
September 5, 2008: Cell
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