Literature DB >> 29967362

Why dystrophin quantification is key in the eteplirsen saga.

Annemieke Aartsma-Rus1, Virginia Arechavala-Gomeza2.   

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Year:  2018        PMID： 29967362     DOI： 10.1038/s41582-018-0033-8

Source DB:  PubMed          Journal:  Nat Rev Neurol        ISSN： 1759-4758            Impact factor:   42.937


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  10 in total

1.  The 10th Oligonucleotide Therapy Approved: Golodirsen for Duchenne Muscular Dystrophy.

Authors:  Annemieke Aartsma-Rus; David R Corey
Journal:  Nucleic Acid Ther       Date:  2020-02-11       Impact factor: 5.486

2.  Exons 45-55 Skipping Using Mutation-Tailored Cocktails of Antisense Morpholinos in the DMD Gene.

Authors:  Yusuke Echigoya; Kenji Rowel Q Lim; Dyanna Melo; Bo Bao; Nhu Trieu; Yoshitaka Mizobe; Rika Maruyama; Kamel Mamchaoui; Jun Tanihata; Yoshitsugu Aoki; Shin'ichi Takeda; Vincent Mouly; William Duddy; Toshifumi Yokota
Journal:  Mol Ther       Date:  2019-07-26       Impact factor: 11.454

3.  Evaluation of Exon Skipping and Dystrophin Restoration in In Vitro Models of Duchenne Muscular Dystrophy.

Authors:  Andrea López-Martínez; Patricia Soblechero-Martín; Virginia Arechavala-Gomeza
Journal:  Methods Mol Biol       Date:  2022

4.  Antisense RNA Therapeutics: A Brief Overview.

Authors:  Virginia Arechavala-Gomeza; Alejandro Garanto
Journal:  Methods Mol Biol       Date:  2022

5.  Endogenous ADAR-mediated RNA editing in non-human primates using stereopure chemically modified oligonucleotides.

Authors:  Prashant Monian; Chikdu Shivalila; Genliang Lu; Mamoru Shimizu; David Boulay; Karley Bussow; Michael Byrne; Adam Bezigian; Arindom Chatterjee; David Chew; Jigar Desai; Frank Favaloro; Jack Godfrey; Andrew Hoss; Naoki Iwamoto; Tomomi Kawamoto; Jayakanthan Kumarasamy; Anthony Lamattina; Amber Lindsey; Fangjun Liu; Richard Looby; Subramanian Marappan; Jake Metterville; Ronelle Murphy; Jeff Rossi; Tom Pu; Bijay Bhattarai; Stephany Standley; Snehlata Tripathi; Hailin Yang; Yuan Yin; Hui Yu; Cong Zhou; Luciano H Apponi; Pachamuthu Kandasamy; Chandra Vargeese
Journal:  Nat Biotechnol       Date:  2022-03-07       Impact factor: 68.164

6.  Introduction and History of the Chemistry of Nucleic Acids Therapeutics.

Authors:  Michael J Gait; Sudhir Agrawal
Journal:  Methods Mol Biol       Date:  2022

7.  Detailed genetic and functional analysis of the hDMDdel52/mdx mouse model.

Authors:  Alper Yavas; Rudie Weij; Maaike van Putten; Eleni Kourkouta; Chantal Beekman; Jukka Puoliväli; Timo Bragge; Toni Ahtoniemi; Jeroen Knijnenburg; Marlies Elisabeth Hoogenboom; Yavuz Ariyurek; Annemieke Aartsma-Rus; Judith van Deutekom; Nicole Datson
Journal:  PLoS One       Date:  2020-12-23       Impact factor: 3.240

Review 8.  RNA Targeting in Inherited Neuromuscular Disorders: Novel Therapeutic Strategies to Counteract Mis-Splicing.

Authors:  Veronica Verdile; Gloria Guizzo; Gabriele Ferrante; Maria Paola Paronetto
Journal:  Cells       Date:  2021-10-22       Impact factor: 6.600

9.  Automated immunofluorescence analysis for sensitive and precise dystrophin quantification in muscle biopsies.

Authors:  Tatyana A Vetter; Stefan Nicolau; Adrienne J Bradley; Emma C Frair; Kevin M Flanigan
Journal:  Neuropathol Appl Neurobiol       Date:  2021-12-28       Impact factor: 6.250

10.  Utrophin modulator drugs as potential therapies for Duchenne and Becker muscular dystrophies.

Authors:  Patricia Soblechero-Martín; Andrea López-Martínez; Laura de la Puente-Ovejero; Ainara Vallejo-Illarramendi; Virginia Arechavala-Gomeza
Journal:  Neuropathol Appl Neurobiol       Date:  2021-06-04       Impact factor: 8.090
  10 in total

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