Literature DB >> 30765324

Micro-dystrophin Genes Bring Hope of an Effective Therapy for Duchenne Muscular Dystrophy.

Kay E Davies1, Simon Guiraud2.   

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Year:  2019        PMID： 30765324      PMCID： PMC6402947          DOI： 10.1016/j.ymthe.2019.01.019

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   11.454


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  16 in total

1.  Very mild muscular dystrophy associated with the deletion of 46% of dystrophin.

Authors:  S B England; L V Nicholson; M A Johnson; S M Forrest; D R Love; E E Zubrzycka-Gaarn; D E Bulman; J B Harris; K E Davies
Journal:  Nature       Date:  1990-01-11       Impact factor: 49.962

2.  Cardiac-Specific Expression of ΔH2-R15 Mini-Dystrophin Normalized All Electrocardiogram Abnormalities and the End-Diastolic Volume in a 23-Month-Old Mouse Model of Duchenne Dilated Cardiomyopathy.

Authors:  Nalinda B Wasala; Jin-Hong Shin; Yi Lai; Yongping Yue; Federica Montanaro; Dongsheng Duan
Journal:  Hum Gene Ther       Date:  2018-03-22       Impact factor: 5.695

3.  Dystrophin's central domain forms a complex filament that becomes disorganized by in-frame deletions.

Authors:  Olivier Delalande; Anne-Elisabeth Molza; Raphael Dos Santos Morais; Angélique Chéron; Émeline Pollet; Céline Raguenes-Nicol; Christophe Tascon; Emmanuel Giudice; Marine Guilbaud; Aurélie Nicolas; Arnaud Bondon; France Leturcq; Nicolas Férey; Marc Baaden; Javier Perez; Pierre Roblin; France Piétri-Rouxel; Jean-François Hubert; Mirjam Czjzek; Elisabeth Le Rumeur
Journal:  J Biol Chem       Date:  2018-03-13       Impact factor: 5.157

4.  Differential effects of dystrophin and utrophin gene transfer in immunocompetent muscular dystrophy (mdx) mice.

Authors:  S Ebihara; G H Guibinga; R Gilbert; J Nalbantoglu; B Massie; G Karpati; B J Petrof
Journal:  Physiol Genomics       Date:  2000-09-08       Impact factor: 3.107

5.  Codon and mRNA sequence optimization of microdystrophin transgenes improves expression and physiological outcome in dystrophic mdx mice following AAV2/8 gene transfer.

Authors:  Helen Foster; Paul S Sharp; Takis Athanasopoulos; Capucine Trollet; Ian R Graham; Keith Foster; Dominic J Wells; George Dickson
Journal:  Mol Ther       Date:  2008-09-02       Impact factor: 11.454

6.  Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy.

Authors:  Yi Lai; Gail D Thomas; Yongping Yue; Hsiao T Yang; Dejia Li; Chun Long; Luke Judge; Brian Bostick; Jeffrey S Chamberlain; Ronald L Terjung; Dongsheng Duan
Journal:  J Clin Invest       Date:  2009-02-23       Impact factor: 14.808

7.  Diaphragm rescue alone prevents heart dysfunction in dystrophic mice.

Authors:  Alastair Crisp; Haifang Yin; Aurelie Goyenvalle; Corinne Betts; Hong M Moulton; Yiqi Seow; Arran Babbs; Thomas Merritt; Amer F Saleh; Michael J Gait; Daniel J Stuckey; Kieran Clarke; Kay E Davies; Matthew J A Wood
Journal:  Hum Mol Genet       Date:  2010-11-09       Impact factor: 6.150

Review 8.  Progress toward Gene Therapy for Duchenne Muscular Dystrophy.

Authors:  Joel R Chamberlain; Jeffrey S Chamberlain
Journal:  Mol Ther       Date:  2017-04-15       Impact factor: 12.910

9.  Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy.

Authors:  Caroline Le Guiner; Laurent Servais; Marie Montus; Thibaut Larcher; Bodvaël Fraysse; Sophie Moullec; Marine Allais; Virginie François; Maeva Dutilleul; Alberto Malerba; Taeyoung Koo; Jean-Laurent Thibaut; Béatrice Matot; Marie Devaux; Johanne Le Duff; Jack-Yves Deschamps; Inès Barthelemy; Stéphane Blot; Isabelle Testault; Karim Wahbi; Stéphane Ederhy; Samia Martin; Philippe Veron; Christophe Georger; Takis Athanasopoulos; Carole Masurier; Federico Mingozzi; Pierre Carlier; Bernard Gjata; Jean-Yves Hogrel; Oumeya Adjali; Fulvio Mavilio; Thomas Voit; Philippe Moullier; George Dickson
Journal:  Nat Commun       Date:  2017-07-25       Impact factor: 14.919

10.  Development of Novel Micro-dystrophins with Enhanced Functionality.

Authors:  Julian N Ramos; Katrin Hollinger; Niclas E Bengtsson; James M Allen; Stephen D Hauschka; Jeffrey S Chamberlain
Journal:  Mol Ther       Date:  2019-02-01       Impact factor: 11.454
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  3 in total

Review 1.  Duchenne muscular dystrophy animal models for high-throughput drug discovery and precision medicine.

Authors:  Nalinda B Wasala; Shi-Jie Chen; Dongsheng Duan
Journal:  Expert Opin Drug Discov       Date:  2020-01-30       Impact factor: 6.098

2.  Serum extracellular vesicles for delivery of CRISPR-CAS9 ribonucleoproteins to modify the dystrophin gene.

Authors:  Nathalie Majeau; Annabelle Fortin-Archambault; Catherine Gérard; Joël Rousseau; Pouiré Yaméogo; Jacques P Tremblay
Journal:  Mol Ther       Date:  2022-05-26       Impact factor: 12.910

Review 3.  Natural products, PGC-1 α , and Duchenne muscular dystrophy.

Authors:  Ipek Suntar; Antoni Sureda; Tarun Belwal; Ana Sanches Silva; Rosa Anna Vacca; Devesh Tewari; Eduardo Sobarzo-Sánchez; Seyed Fazel Nabavi; Samira Shirooie; Ahmad Reza Dehpour; Suowen Xu; Bahman Yousefi; Maryam Majidinia; Maria Daglia; Giuseppe D'Antona; Seyed Mohammad Nabavi
Journal:  Acta Pharm Sin B       Date:  2020-01-08       Impact factor: 11.413
  3 in total

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