Detalhe da pesquisa
1.
Dystrophin rescue by trans-splicing: a strategy for DMD genotypes not eligible for exon skipping approaches.
Nucleic Acids Res
; 41(17): 8391-402, 2013 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-23861443
2.
Muscle function recovery in golden retriever muscular dystrophy after AAV1-U7 exon skipping.
Mol Ther
; 20(11): 2120-33, 2012 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-22968479
3.
[Functional correction and cognitive improvement in dystrophic mice using splice-switching tricyclo-DNA oligomers]. / Un nouvel outil pour le traitement de la myopathie de Duchenne : les tricyclo-ADN.
Med Sci (Paris)
; 31(3): 253-6, 2015 Mar.
Artigo
em Francês
| MEDLINE | ID: mdl-25855277
4.
Evaluating the Impact of Variable Phosphorothioate Content in Tricyclo-DNA Antisense Oligonucleotides in a Duchenne Muscular Dystrophy Mouse Model.
Nucleic Acid Ther
; 29(3): 148-160, 2019 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-31009315
5.
AAV-8 and AAV-9 Vectors Cooperate with Serum Proteins Differently Than AAV-1 and AAV-6.
Mol Ther Methods Clin Dev
; 10: 291-302, 2018 Sep 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-30155509
6.
Efficacy and Safety Profile of Tricyclo-DNA Antisense Oligonucleotides in Duchenne Muscular Dystrophy Mouse Model.
Mol Ther Nucleic Acids
; 8: 144-157, 2017 Sep 15.
Artigo
em Inglês
| MEDLINE | ID: mdl-28918017
7.
Efficient SMN Rescue following Subcutaneous Tricyclo-DNA Antisense Oligonucleotide Treatment.
Mol Ther Nucleic Acids
; 7: 81-89, 2017 Jun 16.
Artigo
em Inglês
| MEDLINE | ID: mdl-28624227
8.
Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers.
Nat Med
; 21(3): 270-5, 2015 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-25642938