Literature DB >> 23609411

Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches.

Rebecca J Fairclough1, Matthew J Wood, Kay E Davies.   

Abstract

Duchenne muscular dystrophy (DMD) is a devastating progressive disease for which there is currently no effective treatment except palliative therapy. There are several promising genetic approaches, including viral delivery of the missing dystrophin gene, read-through of translation stop codons, exon skipping to restore the reading frame and increased expression of the compensatory utrophin gene. The lessons learned from these approaches will be applicable to many other disorders.

Entities:  

Mesh：

Substances：

Year:  2013        PMID： 23609411     DOI： 10.1038/nrg3460

Source DB:  PubMed          Journal:  Nat Rev Genet        ISSN： 1471-0056            Impact factor:   53.242


  48 in total

1.  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

2.  Naturally occurring utrophin correlates with disease severity in Duchenne muscular dystrophy.

Authors:  Kleopas A Kleopa; Anthi Drousiotou; Eleni Mavrikiou; Annita Ormiston; Theodoros Kyriakides
Journal:  Hum Mol Genet       Date:  2006-04-04       Impact factor: 6.150

Review 3.  Utrophin upregulation for treating Duchenne or Becker muscular dystrophy: how close are we?

Authors:  Pedro Miura; Bernard J Jasmin
Journal:  Trends Mol Med       Date:  2006-01-27       Impact factor: 11.951

4.  In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping.

Authors:  Hans A Heemskerk; Christa L de Winter; Sjef J de Kimpe; Petra van Kuik-Romeijn; Niki Heuvelmans; Gerard J Platenburg; Gert-Jan B van Ommen; Judith C T van Deutekom; Annemieke Aartsma-Rus
Journal:  J Gene Med       Date:  2009-03       Impact factor: 4.565

5.  Local dystrophin restoration with antisense oligonucleotide PRO051.

Authors:  Judith C van Deutekom; Anneke A Janson; Ieke B Ginjaar; Wendy S Frankhuizen; Annemieke Aartsma-Rus; Mattie Bremmer-Bout; Johan T den Dunnen; Klaas Koop; Anneke J van der Kooi; Nathalie M Goemans; Sjef J de Kimpe; Peter F Ekhart; Edna H Venneker; Gerard J Platenburg; Jan J Verschuuren; Gert-Jan B van Ommen
Journal:  N Engl J Med       Date:  2007-12-27       Impact factor: 91.245

Review 6.  Cancer syndromes and therapy by stop-codon readthrough.

Authors:  Renata Bordeira-Carriço; Ana Paula Pêgo; Manuel Santos; Carla Oliveira
Journal:  Trends Mol Med       Date:  2012-10-06       Impact factor: 11.951

7.  Low dystrophin levels increase survival and improve muscle pathology and function in dystrophin/utrophin double-knockout mice.

Authors:  Maaike van Putten; Margriet Hulsker; Courtney Young; Vishna D Nadarajah; Hans Heemskerk; Louise van der Weerd; Peter A C 't Hoen; Gert-Jan B van Ommen; Annemieke M Aartsma-Rus
Journal:  FASEB J       Date:  2013-03-04       Impact factor: 5.191

8.  Daily treatment with SMTC1100, a novel small molecule utrophin upregulator, dramatically reduces the dystrophic symptoms in the mdx mouse.

Authors:  Jonathon M Tinsley; Rebecca J Fairclough; Richard Storer; Fraser J Wilkes; Allyson C Potter; Sarah E Squire; Dave S Powell; Anna Cozzoli; Roberta F Capogrosso; Adam Lambert; Francis X Wilson; Stephen P Wren; Annamaria De Luca; Kay E Davies
Journal:  PLoS One       Date:  2011-05-06       Impact factor: 3.240

Review 9.  Gene therapy for severe combined immunodeficiency: are we there yet?

Authors:  Marina Cavazzana-Calvo; Alain Fischer
Journal:  J Clin Invest       Date:  2007-06       Impact factor: 14.808

Review 10.  Pharmacologically targeting the primary defect and downstream pathology in Duchenne muscular dystrophy.

Authors:  Rebecca J Fairclough; Kelly J Perkins; Kay E Davies
Journal:  Curr Gene Ther       Date:  2012-06       Impact factor: 4.391
View more
  99 in total

1.  [Computer experience and further developments in the respiratory function laboratory (author's transl)].

Authors:  R Schindl; K Mayer; K Aigner
Journal:  Med Klin       Date:  1975-11-07

Review 2.  Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials.

Authors:  Yuko Shimizu-Motohashi; Shouta Miyatake; Hirofumi Komaki; Shin'ichi Takeda; Yoshitsugu Aoki
Journal:  Am J Transl Res       Date:  2016-06-15       Impact factor: 4.060

3.  Discovery of serum protein biomarkers in the mdx mouse model and cross-species comparison to Duchenne muscular dystrophy patients.

Authors:  Yetrib Hathout; Ramya L Marathi; Sree Rayavarapu; Aiping Zhang; Kristy J Brown; Haeri Seol; Heather Gordish-Dressman; Sebahattin Cirak; Luca Bello; Kanneboyina Nagaraju; Terry Partridge; Eric P Hoffman; Shin'ichi Takeda; Jean K Mah; Erik Henricson; Craig McDonald
Journal:  Hum Mol Genet       Date:  2014-07-15       Impact factor: 6.150

4.  Herpes simplex viral-vector design for efficient transduction of nonneuronal cells without cytotoxicity.

Authors:  Yoshitaka Miyagawa; Pietro Marino; Gianluca Verlengia; Hiroaki Uchida; William F Goins; Shinichiro Yokota; David A Geller; Osamu Yoshida; Joseph Mester; Justus B Cohen; Joseph C Glorioso
Journal:  Proc Natl Acad Sci U S A       Date:  2015-03-16       Impact factor: 11.205

5.  Population Pharmacokinetics of Vamorolone (VBP15) in Healthy Men and Boys With Duchenne Muscular Dystrophy.

Authors:  Panteleimon D Mavroudis; John van den Anker; Laurie S Conklin; Jesse M Damsker; Eric P Hoffman; Kanneboyina Nagaraju; Paula R Clemens; William J Jusko
Journal:  J Clin Pharmacol       Date:  2019-02-11       Impact factor: 3.126

Review 6.  Modulation of aberrant splicing in human RNA diseases by chemical compounds.

Authors:  Naoyuki Kataoka
Journal:  Hum Genet       Date:  2017-03-31       Impact factor: 4.132

7.  Variable cytoplasmic actin expression impacts the sensitivity of different dystrophin-deficient mdx skeletal muscles to eccentric contraction.

Authors:  Angus Lindsay; William M Southern; Preston M McCourt; Alexie A Larson; James S Hodges; Dawn A Lowe; James M Ervasti
Journal:  FEBS J       Date:  2019-04-11       Impact factor: 5.542

8.  Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA.

Authors:  Chengzu Long; John R McAnally; John M Shelton; Alex A Mireault; Rhonda Bassel-Duby; Eric N Olson
Journal:  Science       Date:  2014-08-14       Impact factor: 47.728

Review 9.  Dystrophin and the two related genetic diseases, Duchenne and Becker muscular dystrophies.

Authors:  Elisabeth Le Rumeur
Journal:  Bosn J Basic Med Sci       Date:  2015-07-20       Impact factor: 3.363

10.  Conserved regions of the DMD 3' UTR regulate translation and mRNA abundance in cultured myotubes.

Authors:  C Aaron Larsen; Michael T Howard
Journal:  Neuromuscul Disord       Date:  2014-05-22       Impact factor: 4.296
View more

北京卡尤迪生物科技股份有限公司 © 2022-2023.