Garcia L - Search Results

1

Full-length dysferlin expression driven by engineered human dystrophic blood derived CD133+ stem cells.

Meregalli M, Navarro C, Sitzia C, Farini A, Montani E, Wein N, Razini P, Beley C, Cassinelli L, Parolini D, Belicchi M, Parazzoli D, Garcia L, Torrente Y. Meregalli M, et al. Among authors: garcia l. FEBS J. 2013 Dec;280(23):6045-60. doi: 10.1111/febs.12523. Epub 2013 Oct 8. FEBS J. 2013. PMID: 24028392 Free article.

2

[Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice].

Benchaouir R, Meregalli M, Farini A, D'Antona G, Belicchi M, Goyenvalle A, Battistelli M, Bresolin N, Bottinelli R, Garcia L, Torrente Y. Benchaouir R, et al. Among authors: garcia l. Med Sci (Paris). 2008 Jan;24(1):99-101. doi: 10.1051/medsci/200824199. Med Sci (Paris). 2008. PMID: 18198122 Free article. French. No abstract available.

3

Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice.

Benchaouir R, Meregalli M, Farini A, D'Antona G, Belicchi M, Goyenvalle A, Battistelli M, Bresolin N, Bottinelli R, Garcia L, Torrente Y. Benchaouir R, et al. Among authors: garcia l. Cell Stem Cell. 2007 Dec 13;1(6):646-57. doi: 10.1016/j.stem.2007.09.016. Cell Stem Cell. 2007. PMID: 18371406 Free article.

4

Efficient bypass of mutations in dysferlin deficient patient cells by antisense-induced exon skipping.

Wein N, Avril A, Bartoli M, Beley C, Chaouch S, Laforêt P, Behin A, Butler-Browne G, Mouly V, Krahn M, Garcia L, Lévy N. Wein N, et al. Among authors: garcia l. Hum Mutat. 2010 Feb;31(2):136-42. doi: 10.1002/humu.21160. Hum Mutat. 2010. PMID: 19953532

5

Combination of myostatin pathway interference and dystrophin rescue enhances tetanic and specific force in dystrophic mdx mice.

Dumonceaux J, Marie S, Beley C, Trollet C, Vignaud A, Ferry A, Butler-Browne G, Garcia L. Dumonceaux J, et al. Among authors: garcia l. Mol Ther. 2010 May;18(5):881-7. doi: 10.1038/mt.2009.322. Epub 2010 Jan 26. Mol Ther. 2010. PMID: 20104211 Free PMC article.

6

Therapeutic exon 'switching' for dysferlinopathies?

Lévy N, Wein N, Barthelemy F, Mouly V, Garcia L, Krahn M, Bartoli M. Lévy N, et al. Among authors: garcia l. Eur J Hum Genet. 2010 Sep;18(9):969-70; author reply 971. doi: 10.1038/ejhg.2010.73. Epub 2010 May 26. Eur J Hum Genet. 2010. PMID: 20512160 Free PMC article. No abstract available.

7

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. Vulin A, et al. Among authors: garcia l. Mol Ther. 2012 Nov;20(11):2120-33. doi: 10.1038/mt.2012.181. Epub 2012 Sep 11. Mol Ther. 2012. PMID: 22968479 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. Rendu J, et al. Among authors: garcia l. Hum Gene Ther. 2013 Jul;24(7):702-13. doi: 10.1089/hum.2013.052. Hum Gene Ther. 2013. PMID: 23805838 Free PMC article.

9

In vitro correction of a pseudoexon-generating deep intronic mutation in LGMD2A by antisense oligonucleotides and modified small nuclear RNAs.

Blázquez L, Aiastui A, Goicoechea M, Martins de Araujo M, Avril A, Beley C, García L, Valcárcel J, Fortes P, López de Munain A. Blázquez L, et al. Among authors: garcia l. Hum Mutat. 2013 Oct;34(10):1387-95. doi: 10.1002/humu.22379. Epub 2013 Aug 7. Hum Mutat. 2013. PMID: 23864287

10

Dysferlin rescue by spliceosome-mediated pre-mRNA trans-splicing targeting introns harbouring weakly defined 3' splice sites.

Philippi S, Lorain S, Beley C, Peccate C, Précigout G, Spuler S, Garcia L. Philippi S, et al. Among authors: garcia l. Hum Mol Genet. 2015 Jul 15;24(14):4049-60. doi: 10.1093/hmg/ddv141. Epub 2015 Apr 22. Hum Mol Genet. 2015. PMID: 25904108

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