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基于RNA的候选药物 - 几个实验室已合成了能够特异性结合DMPK Messenger RNA并导致其降解的小RNA片段。Depending on its size and chemical composition, researchers distinguish several kinds: antisen oligonucleotides, interferential small RNAs (Small Interfering RNA in English, or Sirna), micro-arn ... to allow them to penetrate more effectively inside muscle cells, they were optimized: Del-desiran and Dyne-101 are associated with Fragment of antibodies which will bind to a receiver on the surface of muscle cells (the TFR1转铁接收器）虽然PGN-edoDM1和VX-670与能够越过细胞膜的肽有关。
Steinert病的2024年晚期
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