275 related articles for article (PubMed ID: 23752313)
1. 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. PubMed ID: 23752313
[TBL] [Abstract][Full Text] [Related]
2. Antisense pre-treatment increases gene therapy efficacy in dystrophic muscles.
Peccate C; Mollard A; Le Hir M; Julien L; McClorey G; Jarmin S; Le Heron A; Dickson G; Benkhelifa-Ziyyat S; Piétri-Rouxel F; Wood MJ; Voit T; Lorain S
Hum Mol Genet; 2016 Aug; 25(16):3555-3563. PubMed ID: 27378686
[TBL] [Abstract][Full Text] [Related]
3. Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping.
Goyenvalle A; Vulin A; Fougerousse F; Leturcq F; Kaplan JC; Garcia L; Danos O
Science; 2004 Dec; 306(5702):1796-9. PubMed ID: 15528407
[TBL] [Abstract][Full Text] [Related]
4. 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. PubMed ID: 22968479
[TBL] [Abstract][Full Text] [Related]
5. Engineering exon-skipping vectors expressing U7 snRNA constructs for Duchenne muscular dystrophy gene therapy.
Goyenvalle A; Davies KE
Methods Mol Biol; 2011; 709():179-96. PubMed ID: 21194028
[TBL] [Abstract][Full Text] [Related]
6. Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping.
Goyenvalle A; Babbs A; Wright J; Wilkins V; Powell D; Garcia L; Davies KE
Hum Mol Genet; 2012 Jun; 21(11):2559-71. PubMed ID: 22388933
[TBL] [Abstract][Full Text] [Related]
7. Muscle regeneration affects Adeno Associated Virus 1 mediated transgene transcription.
Mollard A; Peccate C; Forand A; Chassagne J; Julien L; Meunier P; Guesmia Z; Marais T; Bitoun M; Piétri-Rouxel F; Benkhelifa-Ziyyat S; Lorain S
Sci Rep; 2022 Jun; 12(1):9674. PubMed ID: 35690627
[TBL] [Abstract][Full Text] [Related]
8. Combined effect of AAV-U7-induced dystrophin exon skipping and soluble activin Type IIB receptor in mdx mice.
Hoogaars WM; Mouisel E; Pasternack A; Hulmi JJ; Relizani K; Schuelke M; Schirwis E; Garcia L; Ritvos O; Ferry A; 't Hoen PA; Amthor H
Hum Gene Ther; 2012 Dec; 23(12):1269-79. PubMed ID: 22894762
[TBL] [Abstract][Full Text] [Related]
9. Chimeric adeno-associated virus/antisense U1 small nuclear RNA effectively rescues dystrophin synthesis and muscle function by local treatment of mdx mice.
Denti MA; Rosa A; D'Antona G; Sthandier O; De Angelis FG; Nicoletti C; Allocca M; Pansarasa O; Parente V; Musarò A; Auricchio A; Bottinelli R; Bozzoni I
Hum Gene Ther; 2006 May; 17(5):565-74. PubMed ID: 16716113
[TBL] [Abstract][Full Text] [Related]
10. Delivery of AAV2/9-microdystrophin genes incorporating helix 1 of the coiled-coil motif in the C-terminal domain of dystrophin improves muscle pathology and restores the level of α1-syntrophin and α-dystrobrevin in skeletal muscles of mdx mice.
Koo T; Malerba A; Athanasopoulos T; Trollet C; Boldrin L; Ferry A; Popplewell L; Foster H; Foster K; Dickson G
Hum Gene Ther; 2011 Nov; 22(11):1379-88. PubMed ID: 21453126
[TBL] [Abstract][Full Text] [Related]
11. Long-term restoration of cardiac dystrophin expression in golden retriever muscular dystrophy following rAAV6-mediated exon skipping.
Bish LT; Sleeper MM; Forbes SC; Wang B; Reynolds C; Singletary GE; Trafny D; Morine KJ; Sanmiguel J; Cecchini S; Virag T; Vulin A; Beley C; Bogan J; Wilson JM; Vandenborne K; Kornegay JN; Walter GA; Kotin RM; Garcia L; Sweeney HL
Mol Ther; 2012 Mar; 20(3):580-9. PubMed ID: 22146342
[TBL] [Abstract][Full Text] [Related]
12. Dual AAV therapy ameliorates exercise-induced muscle injury and functional ischemia in murine models of Duchenne muscular dystrophy.
Zhang Y; Yue Y; Li L; Hakim CH; Zhang K; Thomas GD; Duan D
Hum Mol Genet; 2013 Sep; 22(18):3720-9. PubMed ID: 23681067
[TBL] [Abstract][Full Text] [Related]
13. [Gene therapy for muscular dystrophy].
Takeda S
No To Hattatsu; 2004 Mar; 36(2):117-23. PubMed ID: 15031985
[TBL] [Abstract][Full Text] [Related]
14. Body-wide gene therapy of Duchenne muscular dystrophy in the mdx mouse model.
Denti MA; Rosa A; D'Antona G; Sthandier O; De Angelis FG; Nicoletti C; Allocca M; Pansarasa O; Parente V; Musarò A; Auricchio A; Bottinelli R; Bozzoni I
Proc Natl Acad Sci U S A; 2006 Mar; 103(10):3758-63. PubMed ID: 16501048
[TBL] [Abstract][Full Text] [Related]
15. In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice.
El Refaey M; Xu L; Gao Y; Canan BD; Adesanya TMA; Warner SC; Akagi K; Symer DE; Mohler PJ; Ma J; Janssen PML; Han R
Circ Res; 2017 Sep; 121(8):923-929. PubMed ID: 28790199
[TBL] [Abstract][Full Text] [Related]
16. Dystrophin Gene-Editing Stability Is Dependent on Dystrophin Levels in Skeletal but Not Cardiac Muscles.
Bengtsson NE; Tasfaout H; Hauschka SD; Chamberlain JS
Mol Ther; 2021 Mar; 29(3):1070-1085. PubMed ID: 33160075
[TBL] [Abstract][Full Text] [Related]
17. Perspective on Adeno-Associated Virus Capsid Modification for Duchenne Muscular Dystrophy Gene Therapy.
Nance ME; Duan D
Hum Gene Ther; 2015 Dec; 26(12):786-800. PubMed ID: 26414293
[TBL] [Abstract][Full Text] [Related]
18. Triple trans-splicing adeno-associated virus vectors capable of transferring the coding sequence for full-length dystrophin protein into dystrophic mice.
Koo T; Popplewell L; Athanasopoulos T; Dickson G
Hum Gene Ther; 2014 Feb; 25(2):98-108. PubMed ID: 24191945
[TBL] [Abstract][Full Text] [Related]
19. Restoration of all dystrophin protein interactions by functional domains in trans does not rescue dystrophy.
Gardner KL; Kearney JA; Edwards JD; Rafael-Fortney JA
Gene Ther; 2006 May; 13(9):744-51. PubMed ID: 16307000
[TBL] [Abstract][Full Text] [Related]
20. Sustained AAV-mediated dystrophin expression in a canine model of Duchenne muscular dystrophy with a brief course of immunosuppression.
Wang Z; Kuhr CS; Allen JM; Blankinship M; Gregorevic P; Chamberlain JS; Tapscott SJ; Storb R
Mol Ther; 2007 Jun; 15(6):1160-6. PubMed ID: 17426713
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]