Literature DB >> 24695683

Exon-skipping therapy: a roadblock, detour, or bump in the road?

Eric P Hoffman1, Elizabeth M McNally.   

Abstract

Exon skipping is a promising therapeutic for Duchenne muscular dystrophy patients, but the road to drug approvals is foggy and may require more early-stage derisking and regulatory guidance.

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Year:  2014        PMID： 24695683      PMCID： PMC4464785          DOI： 10.1126/scitranslmed.3008873

Source DB:  PubMed          Journal:  Sci Transl Med        ISSN： 1946-6234            Impact factor:   17.956


  9 in total

1.  Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide.

Authors:  Susan Fletcher; Kaite Honeyman; Abbie M Fall; Penny L Harding; Russell D Johnsen; Steve D Wilton
Journal:  J Gene Med       Date:  2006-02       Impact factor: 4.565

2.  Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs.

Authors:  Toshifumi Yokota; Qi-Long Lu; Terence Partridge; Masanori Kobayashi; Akinori Nakamura; Shińichi Takeda; Eric Hoffman
Journal:  Ann Neurol       Date:  2009-06       Impact factor: 10.422

3.  Systemic administration of PRO051 in Duchenne's muscular dystrophy.

Authors:  Nathalie M Goemans; Mar Tulinius; Johanna T van den Akker; Brigitte E Burm; Peter F Ekhart; Niki Heuvelmans; Tjadine Holling; Anneke A Janson; Gerard J Platenburg; Jessica A Sipkens; J M Ad Sitsen; Annemieke Aartsma-Rus; Gert-Jan B van Ommen; Gunnar Buyse; Niklas Darin; Jan J Verschuuren; Giles V Campion; Sjef J de Kimpe; Judith C van Deutekom
Journal:  N Engl J Med       Date:  2011-03-23       Impact factor: 91.245

4.  Eteplirsen for the treatment of Duchenne muscular dystrophy.

Authors:  Jerry R Mendell; Louise R Rodino-Klapac; Zarife Sahenk; Kandice Roush; Loren Bird; Linda P Lowes; Lindsay Alfano; Ann Maria Gomez; Sarah Lewis; Janaiah Kota; Vinod Malik; Kim Shontz; Christopher M Walker; Kevin M Flanigan; Marco Corridore; John R Kean; Hugh D Allen; Chris Shilling; Kathleen R Melia; Peter Sazani; Jay B Saoud; Edward M Kaye
Journal:  Ann Neurol       Date:  2013-09-10       Impact factor: 10.422

5.  Exon skipping during splicing of dystrophin mRNA precursor due to an intraexon deletion in the dystrophin gene of Duchenne muscular dystrophy kobe.

Authors:  M Matsuo; T Masumura; H Nishio; T Nakajima; Y Kitoh; T Takumi; J Koga; H Nakamura
Journal:  J Clin Invest       Date:  1991-06       Impact factor: 14.808

6.  Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology.

Authors:  Julia Alter; Fang Lou; Adam Rabinowitz; HaiFang Yin; Jeffrey Rosenfeld; Steve D Wilton; Terence A Partridge; Qi Long Lu
Journal:  Nat Med       Date:  2006-01-29       Impact factor: 53.440

7.  Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study.

Authors:  Sebahattin Cirak; Virginia Arechavala-Gomeza; Michela Guglieri; Lucy Feng; Silvia Torelli; Karen Anthony; Stephen Abbs; Maria Elena Garralda; John Bourke; Dominic J Wells; George Dickson; Matthew J A Wood; Steve D Wilton; Volker Straub; Ryszard Kole; Stephen B Shrewsbury; Caroline Sewry; Jennifer E Morgan; Kate Bushby; Francesco Muntoni
Journal:  Lancet       Date:  2011-07-23       Impact factor: 79.321

8.  Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.

Authors:  Yoshitsugu Aoki; Toshifumi Yokota; Tetsuya Nagata; Akinori Nakamura; Jun Tanihata; Takashi Saito; Stephanie M R Duguez; Kanneboyina Nagaraju; Eric P Hoffman; Terence Partridge; Shin'ichi Takeda
Journal:  Proc Natl Acad Sci U S A       Date:  2012-08-06       Impact factor: 11.205

9.  Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice.

Authors:  Alberto Malerba; Francesca C Thorogood; George Dickson; Ian R Graham
Journal:  Hum Gene Ther       Date:  2009-09       Impact factor: 5.695
  9 in total
  21 in total

1.  Could exon skipping help dystrophic boys to run, hop, and jump?

Authors:  Terence Partridge
Journal:  Mol Ther       Date:  2014-11       Impact factor: 11.454

2.  Modulation of Splicing by Single-Stranded Silencing RNAs.

Authors:  Jing Liu; Jiaxin Hu; Jessica A Hicks; Thazha P Prakash; David R Corey
Journal:  Nucleic Acid Ther       Date:  2015-03-10       Impact factor: 5.486

Review 3.  Genetic diagnosis as a tool for personalized treatment of Duchenne muscular dystrophy.

Authors:  Luca Bello; Elena Pegoraro
Journal:  Acta Myol       Date:  2016-12

4.  DMD genotypes and loss of ambulation in the CINRG Duchenne Natural History Study.

Authors:  Luca Bello; Lauren P Morgenroth; Heather Gordish-Dressman; Eric P Hoffman; Craig M McDonald; Sebahattin Cirak
Journal:  Neurology       Date:  2016-06-24       Impact factor: 9.910

5.  A new therapeutic effect of simvastatin revealed by functional improvement in muscular dystrophy.

Authors:  Nicholas P Whitehead; Min Jeong Kim; Kenneth L Bible; Marvin E Adams; Stanley C Froehner
Journal:  Proc Natl Acad Sci U S A       Date:  2015-09-28       Impact factor: 11.205

Review 6.  Absence of Dystrophin Disrupts Skeletal Muscle Signaling: Roles of Ca2+, Reactive Oxygen Species, and Nitric Oxide in the Development of Muscular Dystrophy.

Authors:  David G Allen; Nicholas P Whitehead; Stanley C Froehner
Journal:  Physiol Rev       Date:  2016-01       Impact factor: 37.312

7.  Longitudinal timed function tests in Duchenne muscular dystrophy: ImagingDMD cohort natural history.

Authors:  Harneet Arora; Rebecca J Willcocks; Donovan J Lott; Ann T Harrington; Claudia R Senesac; Kirsten L Zilke; Michael J Daniels; Dandan Xu; Gihan I Tennekoon; Erika L Finanger; Barry S Russman; Richard S Finkel; William T Triplett; Barry J Byrne; Glenn A Walter; H Lee Sweeney; Krista Vandenborne
Journal:  Muscle Nerve       Date:  2018-07-24       Impact factor: 3.217

8.  DMD/BMD prenatal diagnosis and treatment expectation in a single centre in China for 15 years.

Authors:  Xingjian Zhong; Siying Cui; Lina Liu; Yuxia Yang; Xiangdong Kong
Journal:  BMC Med Genomics       Date:  2021-07-08       Impact factor: 3.063

9.  The use of urinary and kidney SILAM proteomics to monitor kidney response to high dose morpholino oligonucleotides in the mdx mouse.

Authors:  Aiping Zhang; Kitipong Uaesoontrachoon; Conner Shaughnessy; Jharna R Das; Sree Rayavarapu; Kristy J Brown; Patricio E Ray; Kanneboyina Nagaraju; John N van den Anker; Eric P Hoffman; Yetrib Hathout
Journal:  Toxicol Rep       Date:  2015

10.  Improving clinical trial design for Duchenne muscular dystrophy.

Authors:  Luciano Merlini; Patrizia Sabatelli
Journal:  BMC Neurol       Date:  2015-08-26       Impact factor: 2.474
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