Literature DB >> 28346110

FDA Approval of Nusinersen for Spinal Muscular Atrophy Makes 2016 the Year of Splice Modulating Oligonucleotides.

Annemieke Aartsma-Rus1.   

Abstract

Entities:  

Keywords:  antisense oligonucleotide; eteplirsen; nusinersen; spinal muscular atrophy; therapy

Mesh：

Substances：

Year:  2017        PMID： 28346110     DOI： 10.1089/nat.2017.0665

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


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  51 in total

1.  A recurrent COL6A1 pseudoexon insertion causes muscular dystrophy and is effectively targeted by splice-correction therapies.

Authors:  Véronique Bolduc; A Reghan Foley; Herimela Solomon-Degefa; Apurva Sarathy; Sandra Donkervoort; Ying Hu; Grace S Chen; Katherine Sizov; Matthew Nalls; Haiyan Zhou; Sara Aguti; Beryl B Cummings; Monkol Lek; Taru Tukiainen; Jamie L Marshall; Oded Regev; Dina Marek-Yagel; Anna Sarkozy; Russell J Butterfield; Cristina Jou; Cecilia Jimenez-Mallebrera; Yan Li; Corine Gartioux; Kamel Mamchaoui; Valérie Allamand; Francesca Gualandi; Alessandra Ferlini; Eric Hanssen; Steve D Wilton; Shireen R Lamandé; Daniel G MacArthur; Raimund Wagener; Francesco Muntoni; Carsten G Bönnemann
Journal:  JCI Insight       Date:  2019-03-21

Review 2.  Nusinersen: The First Option Beyond Supportive Care for Spinal Muscular Atrophy.

Authors:  Vikas Maharshi; Shazia Hasan
Journal:  Clin Drug Investig       Date:  2017-09       Impact factor: 2.859

Review 3.  Advances in therapy for spinal muscular atrophy: promises and challenges.

Authors:  Ewout J N Groen; Kevin Talbot; Thomas H Gillingwater
Journal:  Nat Rev Neurol       Date:  2018-02-09       Impact factor: 42.937

4.  Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts.

Authors:  Rika Maruyama; Aleksander Touznik; Toshifumi Yokota
Journal:  J Vis Exp       Date:  2018-05-11       Impact factor: 1.355

Review 5.  RNA therapy: Are we using the right molecules?

Authors:  Ai-Ming Yu; Chao Jian; Allan H Yu; Mei-Juan Tu
Journal:  Pharmacol Ther       Date:  2018-12-04       Impact factor: 12.310

Review 6.  Mechanism of Splicing Regulation of Spinal Muscular Atrophy Genes.

Authors:  Ravindra N Singh; Natalia N Singh
Journal:  Adv Neurobiol       Date:  2018

7.  Efficient exon skipping of SGCG mutations mediated by phosphorodiamidate morpholino oligomers.

Authors:  Eugene J Wyatt; Alexis R Demonbreun; Ellis Y Kim; Megan J Puckelwartz; Andy H Vo; Lisa M Dellefave-Castillo; Quan Q Gao; Mariz Vainzof; Rita C M Pavanello; Mayana Zatz; Elizabeth M McNally
Journal:  JCI Insight       Date:  2018-05-03

8.  SMN1 gene copy number analysis for spinal muscular atrophy (SMA) in a Turkish cohort by CODE-SEQ technology, an integrated solution for detection of SMN1 and SMN2 copy numbers and the "2+0" genotype.

Authors:  Ahmet Cevdet Ceylan; Haktan Bağış Erdem; İbrahim Şahin; Meenal Agarwal
Journal:  Neurol Sci       Date:  2020-04-06       Impact factor: 3.307

9.  Targeting of CD38 by the Tumor Suppressor miR-26a Serves as a Novel Potential Therapeutic Agent in Multiple Myeloma.

Authors:  Yi Hu; Huimin Liu; Chuanfeng Fang; Chen Li; Fjorela Xhyliu; Hayley Dysert; Juraj Bodo; Gabriel Habermehl; Benjamin E Russell; Wenjun Li; Marcia Chappell; Xiaofeng Jiang; Sarah L Ondrejka; Eric D Hsi; Jaroslaw P Maciejewski; Qing Yi; Kenneth C Anderson; Nikhil C Munshi; Geyou Ao; Jason N Valent; Jianhong Lin; Jianjun Zhao
Journal:  Cancer Res       Date:  2020-03-19       Impact factor: 12.701

10.  RNA Drugs and RNA Targets for Small Molecules: Principles, Progress, and Challenges.

Authors:  Ai-Ming Yu; Young Hee Choi; Mei-Juan Tu
Journal:  Pharmacol Rev       Date:  2020-10       Impact factor: 25.468
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