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Targeted Exon Skipping in Transgenic hDMD Mice: A Model for Direct Preclinical Screening of Human-Specific Antisense Oligonucleotides
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- Molecular Therapy, 2004, v. 10, n. 2, p. 232, doi. 10.1016/j.ymthe.2004.05.031
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Therapeutic antisense-induced exon skipping in cultured muscle cells from six different DMD patients.
- Published in:
- Human Molecular Genetics, 2003, v. 12, n. 8, p. 907, doi. 10.1093/hmg/ddg100
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- Publication type:
- Article