Zobrazeno 1 - 10
of 675
pro vyhledávání: '"Duchenne muscular dystrophy (DMD)"'
Autor:
Jacob Raiten, Genevieve M. Abd, Shane B. Handelsman, Harshank V. Patel, Jennifer C. Ku, Agata M. Parsons, Jonathan L. Wassink, Sheridan L. Hayes, Juliana Overbay, Yong Li
Publikováno v:
Frontiers in Pharmacology, Vol 15 (2024)
Stem cell therapy has shown immense promise in treating genetic disorders, particularly muscular diseases like Duchenne muscular dystrophy (DMD). This study investigates a novel method to enhance the viability of stem cell transplants in DMD by upreg
Externí odkaz:
https://doaj.org/article/d557c33ec8e84c26b1014570db7484fe
Publikováno v:
BMC Medical Genomics, Vol 16, Iss 1, Pp 1-8 (2023)
Abstract Objective This study is a retrospective analysis of the prenatal genetic diagnosis results of 1408 foetuses at high risk of DMD/BMD to provide information for clinical genetic counselling. Background Duchenne muscular dystrophy (DMD) is a se
Externí odkaz:
https://doaj.org/article/399ffbce177540f29bd43879b2aad1af
Autor:
Brenda L. Wong, Suzanne Summer, Paul S. Horn, Meilan M. Rutter, Irina Rybalsky, Cuixia Tian, Karen C. Shellenbarger, Heidi J. Kalkwarf
Publikováno v:
Journal of Cachexia, Sarcopenia and Muscle, Vol 14, Iss 6, Pp 2804-2812 (2023)
Abstract Introduction Mutations in the 79 exons of the dystrophin gene result in muscle wasting and weakness of varying clinical severity, ranging from severe/typical Duchenne muscular dystrophy (DMD) to intermediate DMD and mild Becker muscular dyst
Externí odkaz:
https://doaj.org/article/d225de2543e6495f8c6f65c5c09752d1
Publikováno v:
Biomedicines, Vol 12, Iss 9, p 1996 (2024)
Despite scientific efforts, there is no cure for Duchenne muscular dystrophy (DMD), a lethal, progressive, X-linked genetic disorder caused by mutations in the dystrophin gene. DMD leads to cardiac and skeletal muscle weakness, resulting in premature
Externí odkaz:
https://doaj.org/article/48139f6dd3da46c4b72470d11cd991e2
Publikováno v:
Frontiers in Genetics, Vol 15 (2024)
Duchenne muscular dystrophy (DMD) is a severe genetic disorder characterized by progressive muscle degeneration, with respiratory and cardiac complications, caused by mutations in the DMD gene, encoding the protein dystrophin. Various DMD mutations r
Externí odkaz:
https://doaj.org/article/548dbc9a648841349fd6438f9df7a584
Autor:
Liubov V. Gushchina, Tatyana A. Vetter, Emma C. Frair, Adrienne J. Bradley, Kelly M. Grounds, Jacob W. Lay, Nianyuan Huang, Aisha Suhaiba, Frederick J. Schnell, Gunnar Hanson, Tabatha R. Simmons, Nicolas Wein, Kevin M. Flanigan
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 30, Iss , Pp 479-492 (2022)
Duchenne muscular dystrophy (DMD) is a devastating muscle-wasting disease that arises due to the loss of dystrophin expression, leading to progressive loss of motor and cardiorespiratory function. Four exon-skipping approaches using antisense phospho
Externí odkaz:
https://doaj.org/article/c58f1dc6613c474f8ea734b55a626d47
Autor:
Maria Siemionow, Katarzyna Budzynska, Kristina Zalants, Paulina Langa, Sonia Brodowska, Krzysztof Siemionow, Ahlke Heydemann
Publikováno v:
Biomedicines, Vol 12, Iss 3, p 586 (2024)
Duchenne Muscular Dystrophy (DMD) is a lethal disease caused by mutation in the dystrophin gene. Currently there is no cure for DMD. We introduced a novel human Dystrophin Expressing Chimeric (DEC) cell therapy of myoblast origin and confirmed the sa
Externí odkaz:
https://doaj.org/article/70f7f53f4f054086affb8663d1da0ecf
Akademický článek
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Akademický článek
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Autor:
Josè Manuel Pioner, Lorenzo Santini, Chiara Palandri, Marianna Langione, Bruno Grandinetti, Silvia Querceto, Daniele Martella, Costanza Mazzantini, Beatrice Scellini, Lucrezia Giammarino, Flavia Lupi, Francesco Mazzarotto, Aoife Gowran, Davide Rovina, Rosaria Santoro, Giulio Pompilio, Chiara Tesi, Camilla Parmeggiani, Michael Regnier, Elisabetta Cerbai, David L. Mack, Corrado Poggesi, Cecilia Ferrantini, Raffaele Coppini
Publikováno v:
Frontiers in Physiology, Vol 14 (2023)
Externí odkaz:
https://doaj.org/article/09df826ed4e8452db5de3039b389a56f