HTS-Compatible Patient-Derived Cell-Based Assay to Identify Small Molecule Modulators of Aberrant Splicing in Myotonic Dystrophy Type 1.
适用于高通量筛选的患者来源细胞检测方法,用于鉴定 1 型强直性肌营养不良症中异常剪接的小分子调节剂
期刊:Curr Chem Genomics
影响因子:0.000
doi:10.2174/1875397301004010009
O'Leary Debra A, Vargas Leonardo, Sharif Orzala, Garcia Michael E, Sigal Yury J, Chow Siu-Kei, Schmedt Christian, Caldwell Jeremy S, Brinker Achim, Engels Ingo H