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Challenges of Assessing Exon 53 Skipping of the Human DMD Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for Duchenne Muscular Dystrophy.

Sarah Engelbeen,Daniel O'Reilly,Davy van de Vijver,Ingrid Verhaart,Maaike van Putten,Vignesh Hariharan,Matthew Hassler,Anastasia Khvorova,Masad J. Damha,Annemieke Aartsma-Rus

Nucleic Acid Therapeutics（2023）

引用 5|浏览19
关键词
efficacy,locked nucleic acid,LNA,preclinical studies,exon skipping
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