Format
Sort by

Send to

Choose Destination

Links from PubMed

Items: 1 to 20 of 118

1.

Emerging genetic therapies to treat Duchenne muscular dystrophy.

Nelson SF, Crosbie RH, Miceli MC, Spencer MJ.

Curr Opin Neurol. 2009 Oct;22(5):532-8. doi: 10.1097/WCO.0b013e32832fd487. Review.

2.

[Mutation-specific treatments for Duchenne muscular dystrophy].

Matsuo M, Takeshima Y.

Brain Nerve. 2009 Aug;61(8):915-22. Review. Japanese.

PMID:
19697880
3.

Exon skipping therapy for Duchenne muscular dystrophy.

Kole R, Krieg AM.

Adv Drug Deliv Rev. 2015 Jun 29;87:104-7. doi: 10.1016/j.addr.2015.05.008. Epub 2015 May 14. Review.

PMID:
25980936
4.

A duchenne muscular dystrophy gene hot spot mutation in dystrophin-deficient cavalier king charles spaniels is amenable to exon 51 skipping.

Walmsley GL, Arechavala-Gomeza V, Fernandez-Fuente M, Burke MM, Nagel N, Holder A, Stanley R, Chandler K, Marks SL, Muntoni F, Shelton GD, Piercy RJ.

PLoS One. 2010 Jan 13;5(1):e8647. doi: 10.1371/journal.pone.0008647.

5.

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.

Cirak S, Arechavala-Gomeza V, Guglieri M, Feng L, Torelli S, Anthony K, Abbs S, Garralda ME, Bourke J, Wells DJ, Dickson G, Wood MJ, Wilton SD, Straub V, Kole R, Shrewsbury SB, Sewry C, Morgan JE, Bushby K, Muntoni F.

Lancet. 2011 Aug 13;378(9791):595-605. doi: 10.1016/S0140-6736(11)60756-3. Epub 2011 Jul 23.

6.

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study.

Kinali M, Arechavala-Gomeza V, Feng L, Cirak S, Hunt D, Adkin C, Guglieri M, Ashton E, Abbs S, Nihoyannopoulos P, Garralda ME, Rutherford M, McCulley C, Popplewell L, Graham IR, Dickson G, Wood MJ, Wells DJ, Wilton SD, Kole R, Straub V, Bushby K, Sewry C, Morgan JE, Muntoni F.

Lancet Neurol. 2009 Oct;8(10):918-28. doi: 10.1016/S1474-4422(09)70211-X. Epub 2009 Aug 25. Erratum in: Lancet Neurol. 2009 Dec;8(12):1083.

7.

[Genetic Diagnosis and Molecular Therapies for Duchenne Muscular Dystrophy].

Takeshima Y.

Rinsho Byori. 2015 Oct;63(10):1194-201. Review. Japanese.

PMID:
26897856
8.

Local dystrophin restoration with antisense oligonucleotide PRO051.

van Deutekom JC, Janson AA, Ginjaar IB, Frankhuizen WS, Aartsma-Rus A, Bremmer-Bout M, den Dunnen JT, Koop K, van der Kooi AJ, Goemans NM, de Kimpe SJ, Ekhart PF, Venneker EH, Platenburg GJ, Verschuuren JJ, van Ommen GJ.

N Engl J Med. 2007 Dec 27;357(26):2677-86.

9.

Read-through strategies for suppression of nonsense mutations in Duchenne/ Becker muscular dystrophy: aminoglycosides and ataluren (PTC124).

Finkel RS.

J Child Neurol. 2010 Sep;25(9):1158-64. doi: 10.1177/0883073810371129. Epub 2010 Jun 2. Review.

10.

Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy.

Aartsma-Rus A, Bremmer-Bout M, Janson AA, den Dunnen JT, van Ommen GJ, van Deutekom JC.

Neuromuscul Disord. 2002 Oct;12 Suppl 1:S71-7.

PMID:
12206800
11.

Gene therapy for muscular dystrophy: lessons learned and path forward.

Mendell JR, Rodino-Klapac L, Sahenk Z, Malik V, Kaspar BK, Walker CM, Clark KR.

Neurosci Lett. 2012 Oct 11;527(2):90-9. doi: 10.1016/j.neulet.2012.04.078. Epub 2012 May 17. Review.

12.

[Frontline studies on Duchenne muscular dystrophy treatment].

Matsuo M.

No To Hattatsu. 2009 Mar;41(2):92-5. Review. Japanese.

PMID:
19517771
13.

Skipping multiple exons of dystrophin transcripts using cocktail antisense oligonucleotides.

Echigoya Y, Yokota T.

Nucleic Acid Ther. 2014 Feb;24(1):57-68. doi: 10.1089/nat.2013.0451. Epub 2013 Dec 31. Review.

PMID:
24380394
14.

Systemic administration of PRO051 in Duchenne's muscular dystrophy.

Goemans NM, Tulinius M, van den Akker JT, Burm BE, Ekhart PF, Heuvelmans N, Holling T, Janson AA, Platenburg GJ, Sipkens JA, Sitsen JM, Aartsma-Rus A, van Ommen GJ, Buyse G, Darin N, Verschuuren JJ, Campion GV, de Kimpe SJ, van Deutekom JC.

N Engl J Med. 2011 Apr 21;364(16):1513-22. doi: 10.1056/NEJMoa1011367. Epub 2011 Mar 23. Erratum in: N Engl J Med. 2011 Oct 6;365(14):1361.

15.

Characterization of a complex Duchenne muscular dystrophy-causing dystrophin gene inversion and restoration of the reading frame by induced exon skipping.

Madden HR, Fletcher S, Davis MR, Wilton SD.

Hum Mutat. 2009 Jan;30(1):22-8. doi: 10.1002/humu.20806.

PMID:
18570328
16.

Screening for antisense modulation of dystrophin pre-mRNA splicing.

Dickson G, Hill V, Graham IR.

Neuromuscul Disord. 2002 Oct;12 Suppl 1:S67-70. Review.

PMID:
12206799
17.

Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63% of patients with Duchenne muscular dystrophy.

Béroud C, Tuffery-Giraud S, Matsuo M, Hamroun D, Humbertclaude V, Monnier N, Moizard MP, Voelckel MA, Calemard LM, Boisseau P, Blayau M, Philippe C, Cossée M, Pagès M, Rivier F, Danos O, Garcia L, Claustres M.

Hum Mutat. 2007 Feb;28(2):196-202.

PMID:
17041910
18.

Biochemical characterization of patients with in-frame or out-of-frame DMD deletions pertinent to exon 44 or 45 skipping.

Anthony K, Arechavala-Gomeza V, Ricotti V, Torelli S, Feng L, Janghra N, Tasca G, Guglieri M, Barresi R, Armaroli A, Ferlini A, Bushby K, Straub V, Ricci E, Sewry C, Morgan J, Muntoni F.

JAMA Neurol. 2014 Jan;71(1):32-40. doi: 10.1001/jamaneurol.2013.4908.

PMID:
24217213
19.

Antisense-induced multiexon skipping for Duchenne muscular dystrophy makes more sense.

Aartsma-Rus A, Janson AA, Kaman WE, Bremmer-Bout M, van Ommen GJ, den Dunnen JT, van Deutekom JC.

Am J Hum Genet. 2004 Jan;74(1):83-92. Epub 2003 Dec 16.

20.

Exon skipping for nonsense mutations in Duchenne muscular dystrophy: too many mutations, too few patients?

Yokota T, Duddy W, Echigoya Y, Kolski H.

Expert Opin Biol Ther. 2012 Sep;12(9):1141-52. doi: 10.1517/14712598.2012.693469. Epub 2012 Jun 1. Review.

PMID:
22650324
Items per page

Supplemental Content

Write to the Help Desk