Efficient exon skipping of SGCG mutations mediated by phosphorodiamidate morpholino oligomers
Tóm tắt
Từ khóa
Tài liệu tham khảo
McNally, 1996, Mild and severe muscular dystrophy caused by a single gamma-sarcoglycan mutation, Am J Hum Genet, 59, 1040
Koenig, 1989, The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion, Am J Hum Genet, 45, 498
Clayton, 2014, Antisense Oligonucleotide-mediated Suppression of Muscle Glycogen Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease, Mol Ther Nucleic Acids, 3, 10.1038/mtna.2014.57
Morcos, 2008, Vivo-Morpholinos: a non-peptide transporter delivers Morpholinos into a wide array of mouse tissues, BioTechniques, 45, 613, 10.2144/000113005
Kim, 2016, Direct reprogramming of urine-derived cells with inducible MyoD for modeling human muscle disease, Skelet Muscle, 6, 10.1186/s13395-016-0103-9
Sardone, 2017, Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease, Molecules, 22, 10.3390/molecules22040563
Flicek, 2013, Ensembl 2013, Nucleic Acids Res, 41, D48