Naohiro Takemoto on Muck Rack

Naohiro Takemoto

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Invention of an oral medication for cardiac Fabry disease caused by RNA mis-splicing

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By Yongyong Yang, Shijing Liang, Moriah R. Arnold, Tomonari Awaya, Kei Iida, Megumu Saito, Dau-Ming Niu, Masatoshi Hagiwara, Teruo Sawada, Kentoku Gotanda, Toshiharu Noji, Naohiro Takemoto
| Science Magazine Verified
Research Article DISEASES AND DISORDERS INTRODUCTION Genetic disorders caused by RNA splicing defects have attracted increasing attention (1, 2). Many pathogenic variants affect splicing without altering coding sequences, leading to mis-splicing events that produce non-functional proteins or reduced gene expression.
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