Literature DB >> 27863231

Exon Skipping Therapy.

Courtney S Young1, April D Pyle2.   

Abstract

Exondys 51 is the first therapy for Duchenne muscular dystrophy (DMD) to have been granted accelerated approval by the FDA. Approval was granted based on using dystrophin expression as a surrogate marker. Exondys 51 targets DMD exon 51 for skipping to restore the reading frame for 13% of Duchenne patients. Published by Elsevier Inc.

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Year:  2016        PMID： 27863231     DOI： 10.1016/j.cell.2016.10.050

Source DB:  PubMed          Journal:  Cell        ISSN： 0092-8674            Impact factor:   41.582


  9 in total

1.  SU9516 Increases α7β1 Integrin and Ameliorates Disease Progression in the mdx Mouse Model of Duchenne Muscular Dystrophy.

Authors:  Apurva Sarathy; Ryan D Wuebbles; Tatiana M Fontelonga; Ashley R Tarchione; Lesley A Mathews Griner; Dante J Heredia; Andreia M Nunes; Suzann Duan; Paul D Brewer; Tyler Van Ry; Grant W Hennig; Thomas W Gould; Andrés E Dulcey; Amy Wang; Xin Xu; Catherine Z Chen; Xin Hu; Wei Zheng; Noel Southall; Marc Ferrer; Juan Marugan; Dean J Burkin
Journal:  Mol Ther       Date:  2017-04-05       Impact factor: 11.454

Review 2.  Splicing Modulation as a Promising Therapeutic Strategy for Lysosomal Storage Disorders: The Mucopolysaccharidoses Example.

Authors:  Juliana Inês Santos; Mariana Gonçalves; Liliana Matos; Luciana Moreira; Sofia Carvalho; Maria João Prata; Maria Francisca Coutinho; Sandra Alves
Journal:  Life (Basel)       Date:  2022-04-19

Review 3.  Targeting Splicing in the Treatment of Human Disease.

Authors:  Marc Suñé-Pou; Silvia Prieto-Sánchez; Sofía Boyero-Corral; Cristina Moreno-Castro; Younes El Yousfi; Josep Mª Suñé-Negre; Cristina Hernández-Munain; Carlos Suñé
Journal:  Genes (Basel)       Date:  2017-02-24       Impact factor: 4.096

4.  Enhancing the cytotoxicity of chemoradiation with radiation-guided delivery of anti-MGMT morpholino oligonucleotides in non-methylated solid tumors.

Authors:  P Ambady; Y J Wu; J M Walker; C Kersch; M A Pagel; R L Woltjer; R Fu; L L Muldoon; E A Neuwelt
Journal:  Cancer Gene Ther       Date:  2017-07-28       Impact factor: 5.987

Review 5.  Advances in the delivery of RNA therapeutics: from concept to clinical reality.

Authors:  James C Kaczmarek; Piotr S Kowalski; Daniel G Anderson
Journal:  Genome Med       Date:  2017-06-27       Impact factor: 11.117

Review 6.  Diversification of the muscle proteome through alternative splicing.

Authors:  Kiran Nakka; Claudia Ghigna; Davide Gabellini; F Jeffrey Dilworth
Journal:  Skelet Muscle       Date:  2018-03-06       Impact factor: 4.912

7.  CRISPR/Cas9-generated mouse model of Duchenne muscular dystrophy recapitulating a newly identified large 430 kb deletion in the human DMD gene.

Authors:  Tatiana V Egorova; Evgenia D Zotova; Denis A Reshetov; Anna V Polikarpova; Svetlana G Vassilieva; Dmitry V Vlodavets; Alexey A Gavrilov; Sergey V Ulianov; Vladimir L Buchman; Alexei V Deykin
Journal:  Dis Model Mech       Date:  2019-04-25       Impact factor: 5.758

8.  Dual Fluorescence Splicing Reporter Minigene Identifies an Antisense Oligonucleotide to Skip Exon v8 of the CD44 Gene.

Authors:  Sachiyo Fukushima; Manal Farea; Kazuhiro Maeta; Abdul Qawee Mahyoob Rani; Kazumichi Fujioka; Hisahide Nishio; Masafumi Matsuo
Journal:  Int J Mol Sci       Date:  2020-11-30       Impact factor: 5.923

9.  The Splicing of the Mitochondrial Calcium Uniporter Genuine Activator MICU1 Is Driven by RBFOX2 Splicing Factor during Myogenic Differentiation.

Authors:  Denis Vecellio Reane; Cristina Cerqua; Sabrina Sacconi; Leonardo Salviati; Eva Trevisson; Anna Raffaello
Journal:  Int J Mol Sci       Date:  2022-02-24       Impact factor: 6.208
  9 in total

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