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Results: 1 to 20 of 79

1.

Influence of immune responses in gene/stem cell therapies for muscular dystrophies.

Farini A, Sitzia C, Erratico S, Meregalli M, Torrente Y.

Biomed Res Int. 2014;2014:818107. doi: 10.1155/2014/818107. Epub 2014 May 19.

PMID:
24959590
[PubMed - in process]
Free PMC Article
2.

Development of therapeutic splice-switching oligonucleotides.

Disterer P, Kryczka A, Liu Y, Badi YE, Wong JJ, Owen JS, Khoo B.

Hum Gene Ther. 2014 Jul;25(7):587-98. doi: 10.1089/hum.2013.234. Epub 2014 Jun 19.

PMID:
24826963
[PubMed - in process]
Free PMC Article
3.

Immune Responses to AAV-Vectors, the Glybera Example from Bench to Bedside.

Ferreira V, Petry H, Salmon F.

Front Immunol. 2014 Mar 3;5:82. doi: 10.3389/fimmu.2014.00082. eCollection 2014.

PMID:
24624131
[PubMed]
Free PMC Article
4.

Micro-minicircle Gene Therapy: Implications of Size on Fermentation, Complexation, Shearing Resistance, and Expression.

Stenler S, Wiklander OP, Badal-Tejedor M, Turunen J, Nordin JZ, Hallengärd D, Wahren B, Andaloussi SE, Rutland MW, Smith CE, Lundin KE, Blomberg P.

Mol Ther Nucleic Acids. 2014 Jan 7;2:e140. doi: 10.1038/mtna.2013.67.

PMID:
24399204
[PubMed]
Free PMC Article
5.

Current Challenges and Future Directions in Recombinant AAV-Mediated Gene Therapy of Duchenne Muscular Dystrophy.

Okada T, Takeda S.

Pharmaceuticals (Basel). 2013 Jun 27;6(7):813-36. doi: 10.3390/ph6070813.

PMID:
24276316
[PubMed]
Free PMC Article
6.

Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approaches.

Lorain S, Peccate C, Le Hir M, Griffith G, Philippi S, Précigout G, Mamchaoui K, Jollet A, Voit T, Garcia L.

Nucleic Acids Res. 2013 Sep;41(17):8391-402. doi: 10.1093/nar/gkt621. Epub 2013 Jul 16.

PMID:
23861443
[PubMed - indexed for MEDLINE]
Free PMC Article
7.

Exon skipping and gene transfer restore dystrophin expression in human induced pluripotent stem cells-cardiomyocytes harboring DMD mutations.

Dick E, Kalra S, Anderson D, George V, Ritso M, Laval SH, Barresi R, Aartsma-Rus A, Lochmüller H, Denning C.

Stem Cells Dev. 2013 Oct 15;22(20):2714-24. doi: 10.1089/scd.2013.0135. Epub 2013 Jul 5.

PMID:
23829870
[PubMed - indexed for MEDLINE]
Free PMC Article
8.

Exon skipping as a therapeutic strategy applied to an RYR1 mutation with pseudo-exon inclusion causing a severe core myopathy.

Rendu J, Brocard J, Denarier E, Monnier N, Piétri-Rouxel F, Beley C, Roux-Buisson N, Gilbert-Dussardier B, Perez MJ, Romero N, Garcia L, Lunardi J, Fauré J, Fourest-Lieuvin A, Marty I.

Hum Gene Ther. 2013 Jul;24(7):702-13. doi: 10.1089/hum.2013.052.

PMID:
23805838
[PubMed - indexed for MEDLINE]
Free PMC Article
9.

AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy.

Le Hir M, Goyenvalle A, Peccate C, Précigout G, Davies KE, Voit T, Garcia L, Lorain S.

Mol Ther. 2013 Aug;21(8):1551-8. doi: 10.1038/mt.2013.121. Epub 2013 Jun 11.

PMID:
23752313
[PubMed - indexed for MEDLINE]
Free PMC Article
10.

Rescue of cardiomyopathy through U7snRNA-mediated exon skipping in Mybpc3-targeted knock-in mice.

Gedicke-Hornung C, Behrens-Gawlik V, Reischmann S, Geertz B, Stimpel D, Weinberger F, Schlossarek S, Précigout G, Braren I, Eschenhagen T, Mearini G, Lorain S, Voit T, Dreyfus PA, Garcia L, Carrier L.

EMBO Mol Med. 2013 Jul;5(7):1060-77. doi: 10.1002/emmm.201202168. Epub 2013 May 29.

PMID:
23716398
[PubMed - indexed for MEDLINE]
Free PMC Article
11.

Splicing therapy for neuromuscular disease.

Douglas AG, Wood MJ.

Mol Cell Neurosci. 2013 Sep;56:169-85. doi: 10.1016/j.mcn.2013.04.005. Epub 2013 Apr 28. Review.

PMID:
23631896
[PubMed - indexed for MEDLINE]
Free PMC Article
12.

Gene and cell-mediated therapies for muscular dystrophy.

Konieczny P, Swiderski K, Chamberlain JS.

Muscle Nerve. 2013 May;47(5):649-63. doi: 10.1002/mus.23738. Epub 2013 Mar 29. Review.

PMID:
23553671
[PubMed - indexed for MEDLINE]
Free PMC Article
13.

Exon-skipping strategy by ratio modulation between cytoprotective versus pro-apoptotic clusterin forms increased sensitivity of LNCaP to cell death.

Essabbani A, Garcia L, Zonetti MJ, Fisco T, Pucci S, Chiocchia G.

PLoS One. 2013;8(2):e54920. doi: 10.1371/journal.pone.0054920. Epub 2013 Feb 13.

PMID:
23418433
[PubMed - indexed for MEDLINE]
Free PMC Article
14.

The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery.

Eckenfelder A, Tordo J, Babbs A, Davies KE, Goyenvalle A, Danos O.

Mol Ther Nucleic Acids. 2012 Jun 26;1:e31. doi: 10.1038/mtna.2012.24.

PMID:
23344083
[PubMed]
Free PMC Article
15.

Antisense Oligonucleotide (AON)-based Therapy for Leber Congenital Amaurosis Caused by a Frequent Mutation in CEP290.

Collin RW, den Hollander AI, van der Velde-Visser SD, Bennicelli J, Bennett J, Cremers FP.

Mol Ther Nucleic Acids. 2012 Mar 27;1:e14. doi: 10.1038/mtna.2012.3.

PMID:
23343883
[PubMed]
Free PMC Article
16.

Exon 45 skipping through U1-snRNA antisense molecules recovers the Dys-nNOS pathway and muscle differentiation in human DMD myoblasts.

Cazzella V, Martone J, Pinnarò C, Santini T, Twayana SS, Sthandier O, D'Amico A, Ricotti V, Bertini E, Muntoni F, Bozzoni I.

Mol Ther. 2012 Nov;20(11):2134-42. doi: 10.1038/mt.2012.178. Epub 2012 Sep 11.

PMID:
22968481
[PubMed - indexed for MEDLINE]
Free PMC Article
17.

Muscle function recovery in golden retriever muscular dystrophy after AAV1-U7 exon skipping.

Vulin A, Barthélémy I, Goyenvalle A, Thibaud JL, Beley C, Griffith G, Benchaouir R, le Hir M, Unterfinger Y, Lorain S, Dreyfus P, Voit T, Carlier P, Blot S, Garcia L.

Mol Ther. 2012 Nov;20(11):2120-33. doi: 10.1038/mt.2012.181. Epub 2012 Sep 11.

PMID:
22968479
[PubMed - indexed for MEDLINE]
Free PMC Article
18.

MyD88 signaling in B cells regulates the production of Th1-dependent antibodies to AAV.

Sudres M, Ciré S, Vasseur V, Brault L, Da Rocha S, Boisgérault F, Le Bec C, Gross DA, Blouin V, Ryffel B, Galy A.

Mol Ther. 2012 Aug;20(8):1571-81. doi: 10.1038/mt.2012.101. Epub 2012 May 29.

PMID:
22643865
[PubMed - indexed for MEDLINE]
Free PMC Article
19.

MRI roadmap-guided transendocardial delivery of exon-skipping recombinant adeno-associated virus restores dystrophin expression in a canine model of Duchenne muscular dystrophy.

Barbash IM, Cecchini S, Faranesh AZ, Virag T, Li L, Yang Y, Hoyt RF, Kornegay JN, Bogan JR, Garcia L, Lederman RJ, Kotin RM.

Gene Ther. 2013 Mar;20(3):274-82. doi: 10.1038/gt.2012.38. Epub 2012 May 3.

PMID:
22551778
[PubMed - indexed for MEDLINE]
Free PMC Article
20.

Impaired adaptive response to mechanical overloading in dystrophic skeletal muscle.

Joanne P, Hourdé C, Ochala J, Caudéran Y, Medja F, Vignaud A, Mouisel E, Hadj-Said W, Arandel L, Garcia L, Goyenvalle A, Mounier R, Zibroba D, Sakamoto K, Butler-Browne G, Agbulut O, Ferry A.

PLoS One. 2012;7(4):e35346. doi: 10.1371/journal.pone.0035346. Epub 2012 Apr 12. Erratum in: PLoS One. 2013;8(6). doi:10.1371/annotation/2c0f8405-a2cc-4240-8e1b-117d3ac1a154. Sakamato, Kei [name corrected to Sakamoto, Kei].

PMID:
22511986
[PubMed - indexed for MEDLINE]
Free PMC Article

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