Literature DB >> 32043902

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

Annemieke Aartsma-Rus1, David R Corey2,3.   

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Year:  2020        PMID： 32043902      PMCID： PMC7133412          DOI： 10.1089/nat.2020.0845

Source DB:  PubMed          Journal:  Nucleic Acid Ther        ISSN： 2159-3337            Impact factor:   5.486


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  14 in total

1.  Why dystrophin quantification is key in the eteplirsen saga.

Authors:  Annemieke Aartsma-Rus; Virginia Arechavala-Gomeza
Journal:  Nat Rev Neurol       Date:  2018-08       Impact factor: 42.937

2.  Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production.

Authors:  Jay S Charleston; Frederick J Schnell; Johannes Dworzak; Cas Donoghue; Sarah Lewis; Lei Chen; G David Young; Anthony J Milici; Jon Voss; Uditha DeAlwis; Bruce Wentworth; Louise R Rodino-Klapac; Zarife Sahenk; Diane Frank; Jerry R Mendell
Journal:  Neurology       Date:  2018-05-11       Impact factor: 9.910

Review 3.  Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations.

Authors:  Annemieke Aartsma-Rus; Ivo Fokkema; Jan Verschuuren; Ieke Ginjaar; Judith van Deutekom; Gert-Jan van Ommen; Johan T den Dunnen
Journal:  Hum Mutat       Date:  2009-03       Impact factor: 4.878

4.  The TREAT-NMD DMD Global Database: analysis of more than 7,000 Duchenne muscular dystrophy mutations.

Authors:  Catherine L Bladen; David Salgado; Soledad Monges; Maria E Foncuberta; Kyriaki Kekou; Konstantina Kosma; Hugh Dawkins; Leanne Lamont; Anna J Roy; Teodora Chamova; Velina Guergueltcheva; Sophelia Chan; Lawrence Korngut; Craig Campbell; Yi Dai; Jen Wang; Nina Barišić; Petr Brabec; Jaana Lahdetie; Maggie C Walter; Olivia Schreiber-Katz; Veronika Karcagi; Marta Garami; Venkatarman Viswanathan; Farhad Bayat; Filippo Buccella; En Kimura; Zaïda Koeks; Janneke C van den Bergen; Miriam Rodrigues; Richard Roxburgh; Anna Lusakowska; Anna Kostera-Pruszczyk; Janusz Zimowski; Rosário Santos; Elena Neagu; Svetlana Artemieva; Vedrana Milic Rasic; Dina Vojinovic; Manuel Posada; Clemens Bloetzer; Pierre-Yves Jeannet; Franziska Joncourt; Jordi Díaz-Manera; Eduard Gallardo; A Ayşe Karaduman; Haluk Topaloğlu; Rasha El Sherif; Angela Stringer; Andriy V Shatillo; Ann S Martin; Holly L Peay; Matthew I Bellgard; Jan Kirschner; Kevin M Flanigan; Volker Straub; Kate Bushby; Jan Verschuuren; Annemieke Aartsma-Rus; Christophe Béroud; Hanns Lochmüller
Journal:  Hum Mutat       Date:  2015-03-17       Impact factor: 4.878

Review 5.  The importance of genetic diagnosis for Duchenne muscular dystrophy.

Authors:  Annemieke Aartsma-Rus; Ieke B Ginjaar; Kate Bushby
Journal:  J Med Genet       Date:  2016-01-11       Impact factor: 6.318

6.  FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga.

Authors:  Annemieke Aartsma-Rus; Arthur M Krieg
Journal:  Nucleic Acid Ther       Date:  2016-12-08       Impact factor: 5.486

Review 7.  Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues.

Authors:  Annemieke Aartsma-Rus; Volker Straub; Robert Hemmings; Manuel Haas; Gabriele Schlosser-Weber; Violeta Stoyanova-Beninska; Eugenio Mercuri; Francesco Muntoni; Bruno Sepodes; Elizabeth Vroom; Pavel Balabanov
Journal:  Nucleic Acid Ther       Date:  2017-08-10       Impact factor: 5.486

8.  Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy.

Authors:  Navid Khan; Helen Eliopoulos; Lixin Han; T Bernard Kinane; Linda P Lowes; Jerry R Mendell; Heather Gordish-Dressman; Erik K Henricson; Craig M McDonald
Journal:  J Neuromuscul Dis       Date:  2019

9.  Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy.

Authors:  Jerry R Mendell; Nathalie Goemans; Linda P Lowes; Lindsay N Alfano; Katherine Berry; James Shao; Edward M Kaye; Eugenio Mercuri
Journal:  Ann Neurol       Date:  2016-01-08       Impact factor: 10.422

10.  Identifying and Avoiding tcDNA-ASO Sequence-Specific Toxicity for the Development of DMD Exon 51 Skipping Therapy.

Authors:  Philippine Aupy; Lucía Echevarría; Karima Relizani; Faouzi Zarrouki; Adrian Haeberli; Marek Komisarski; Thomas Tensorer; Grégory Jouvion; Fedor Svinartchouk; Luis Garcia; Aurélie Goyenvalle
Journal:  Mol Ther Nucleic Acids       Date:  2019-11-27       Impact factor: 8.886
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  31 in total

Review 1.  Antisense technology: an overview and prospectus.

Authors:  Stanley T Crooke; Brenda F Baker; Rosanne M Crooke; Xue-Hai Liang
Journal:  Nat Rev Drug Discov       Date:  2021-03-24       Impact factor: 84.694

2.  Modeling Splicing Variants Amenable to Antisense Therapy by Use of CRISPR-Cas9-Based Gene Editing in HepG2 Cells.

Authors:  Arístides López-Márquez; Ainhoa Martínez-Pizarro; Belén Pérez; Eva Richard; Lourdes R Desviat
Journal:  Methods Mol Biol       Date:  2022

3.  A two-residue nascent-strand steric gate controls synthesis of 2'-O-methyl- and 2'-O-(2-methoxyethyl)-RNA.

Authors:  Niklas Freund; Alexander I Taylor; Sebastian Arangundy-Franklin; Nithya Subramanian; Sew-Yeu Peak-Chew; Amy M Whitaker; Bret D Freudenthal; Mikhail Abramov; Piet Herdewijn; Philipp Holliger
Journal:  Nat Chem       Date:  2022-10-13       Impact factor: 24.274

4.  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

5.  Fluorescent base analogues in gapmers enable stealth labeling of antisense oligonucleotide therapeutics.

Authors:  Jesper R Nilsson; Tom Baladi; Audrey Gallud; Dženita Baždarević; Malin Lemurell; Elin K Esbjörner; L Marcus Wilhelmsson; Anders Dahlén
Journal:  Sci Rep       Date:  2021-05-31       Impact factor: 4.379

6.  Low human dystrophin levels prevent cardiac electrophysiological and structural remodelling in a Duchenne mouse model.

Authors:  Gerard A Marchal; Maaike van Putten; Arie O Verkerk; Simona Casini; Kayleigh Putker; Shirley C M van Amersfoorth; Annemieke Aartsma-Rus; Elisabeth M Lodder; Carol Ann Remme
Journal:  Sci Rep       Date:  2021-05-07       Impact factor: 4.379

7.  Matrilineal analysis of mutations in the DMD gene in a multigenerational South Indian cohort using DMD gene panel sequencing.

Authors:  Arun Shastry; Sankaramoorthy Aravind; Meeta Sunil; Keerthi Ramesh; Berty Ashley; Nithyanandan T; Vedam L Ramprasad; Ravi Gupta; Somasekar Seshagiri; Upendra Nongthomba; Sameer Phalke
Journal:  Mol Genet Genomic Med       Date:  2021-05-07       Impact factor: 2.183

Review 8.  Programmable technologies to manipulate gene expression at the RNA level.

Authors:  Huachun Liu; Simone Rauch; Bryan C Dickinson
Journal:  Curr Opin Chem Biol       Date:  2021-04-27       Impact factor: 8.972

Review 9.  Small nucleic acids and the path to the clinic for anti-CRISPR.

Authors:  Christopher L Barkau; Daniel O'Reilly; Seth B Eddington; Masad J Damha; Keith T Gagnon
Journal:  Biochem Pharmacol       Date:  2021-02-27       Impact factor: 6.100

10.  2'-O-(N-(Aminoethyl)carbamoyl)methyl Modification Allows for Lower Phosphorothioate Content in Splice-Switching Oligonucleotides with Retained Activity.

Authors:  Dmytro Honcharenko; Cristina S J Rocha; Karin E Lundin; Jyotirmoy Maity; Stefan Milton; Ulf Tedebark; Merita Murtola; Malgorzata Honcharenko; Andis Slaitas; C I Edvard Smith; Rula Zain; Roger Strömberg
Journal:  Nucleic Acid Ther       Date:  2022-03-02       Impact factor: 4.244
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